Assessing Pharmacogenomic loci Associated with the Pharmacokinetics of Vamorolone in Boys with Duchenne Muscular Dystrophy.

Autor: Li X; Department of Pharmaceutical Sciences, University at Buffalo, State University of New York, Buffalo, NY, USA., Sabbatini D; Department of Neurosciences, University of Padova, Padua, Italy., Pegoraro E; Department of Neurosciences, University of Padova, Padua, Italy., Bello L; Department of Neurosciences, University of Padova, Padua, Italy., Clemens P; Department of Neurology, University of Pittsburgh, Pittsburgh, PA, USA., Guglieri M; John Walton Centre for Neuromuscular Disease, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle-upon-Tyne, UK., van den Anker J; Division of Clinical Pharmacology, Children's National Hospital, Washington, DC, USA.; ReveraGen BioPharma, Rockville, MD, USA., Damsker J; ReveraGen BioPharma, Rockville, MD, USA., McCall J; ReveraGen BioPharma, Rockville, MD, USA., Dang UJ; Department of Health Sciences, Carleton University, Ottawa, Canada., Hoffman EP; ReveraGen BioPharma, Rockville, MD, USA.; Department of Pharmaceutical Sciences, Binghamton University, State University of New York, Binghamton, NY, USA., Jusko WJ; Department of Pharmaceutical Sciences, University at Buffalo, State University of New York, Buffalo, NY, USA.

Publikováno v: Journal of clinical pharmacology [J Clin Pharmacol] 2024 Sep; Vol. 64 (9), pp. 1130-1140. Date of Electronic Publication: 2024 Apr 29.

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.

Autor: Dang UJ; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Damsker JM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Guglieri M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Clemens PR; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Perlman SJ; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Smith EC; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Horrocks I; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Finkel RS; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Mah JK; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Deconinck N; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Goemans NM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Haberlová J; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Straub V; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Mengle-Gaw L; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Schwartz BD; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Harper A; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Shieh PB; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., De Waele L; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Castro D; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Yang ML; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Ryan MM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., McDonald CM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Tulinius M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Webster RI; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Mcmillan HJ; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Kuntz N; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Rao VK; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Baranello G; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Spinty S; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Childs AM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Sbrocchi AM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Selby KA; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Monduy M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Nevo Y; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Vilchez JJ; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Nascimento-Osorio A; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Niks EH; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., De Groot IJM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Katsalouli M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Van Den Anker JN; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Ward LM; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Leinonen M; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., D'Alessandro AL; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton., Hoffman EP; From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; 'P&A Kyriakou' Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton.

Publikováno v: Neurology [Neurology] 2024 Mar 12; Vol. 102 (5), pp. e208112. Date of Electronic Publication: 2024 Feb 09.

Findings from the Longitudinal CINRG Becker Natural History Study.

Autor: Clemens PR; University of Pittsburgh, Pittsburgh, PA, USA., Gordish-Dressman H; Children's National Medical Center, Washington, DC, USA., Niizawa G; University of Pittsburgh, Pittsburgh, PA, USA., Gorni K; Hoffman-La Roche Inc., Basel, Switzerland., Guglieri M; Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Connolly AM; Nationwide Children's Hospital, Columbus, OH, USA., Wicklund M; University of Texas Health Science Center at San Antonio, San Antonio, TX, USA., Bertorini T; University of Tennessee, Memphis, TN, USA., Mah J; Cumming School of Medicine, University of Calgary, Calgary, Canada., Thangarajh M; Virginia Commonwealth University, Richmond, VA, USA., Smith EC; Duke University, Durham, NC, USA., Kuntz NL; Lurie Children's Hospital, Chicago, IL, USA., McDonald CM; University of California, Davis, Sacramento, CA, USA., Henricson E; University of California, Davis, Sacramento, CA, USA., Upadhyayula S; Emory University, Atlanta, GA, USA., Byrne B; University of Florida, Gainesville, FL, USA., Manousakis G; University of Minnesota, Minneapolis, MN, USA., Harper A; Virginia Commonwealth University, Richmond, VA, USA., Iannaccone S; University of Texas Southwestern Medical Center, Dallas, TX, USA., Dang UJ; Carleton University, Ottawa, Canada.

Publikováno v: Journal of neuromuscular diseases [J Neuromuscul Dis] 2024; Vol. 11 (1), pp. 201-212.

Risk factors associated with prevalent vertebral fractures in Duchenne muscular dystrophy.

Autor: Phung K; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., McAdam L; Holland Bloorview Kids Rehabilitation Hospital, Toronto, ON, Canada., Ma J; Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada., McMillan HJ; Montreal Children's Hospital, McGill University, Montreal, QC, Canada., Jackowski S; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., Scharke M; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., Matzinger MA; Department of Medical Imaging, University of Ottawa, Ottawa, ON, Canada., Shenouda N; Department of Medical Imaging, University of Ottawa, Ottawa, ON, Canada., Koujok K; Department of Medical Imaging, University of Ottawa, Ottawa, ON, Canada., Jaremko JL; Department of Radiology and Diagnostic Imaging, University of Alberta, Edmonton, AB, Canada., Smit K; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada., Walker S; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., Hartigan C; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., Khan N; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., Konji VN; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., MacLeay L; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., Page M; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., Sykes E; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada., Robinson ME; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada., Alos N; CHU Sainte-Justine, University of Montreal, Montreal, QC, Canada., Cummings EA; IWK Health Centre, Dalhousie University, Halifax, NS, Canada., Ho J; Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada., Sbrocchi AM; Montreal Children's Hospital, McGill University, Montreal, QC, Canada., Stein R; London Health Sciences Centre, Western University, London, ON, Canada., Saleh D; Kingston Health Sciences Centre, Queen's University, Kingston, ON, Canada., Craven BC; Department of Medicine, Temerty Faculty of Medicine, University Health Network, University of Toronto, Toronto, ON, Canada., Dang UJ; Department of Health Sciences, Carleton University, Ottawa, ON, Canada., Siminoski K; Department of Radiology and Diagnostic Imaging, University of Alberta, Edmonton, AB, Canada., Rauch F; Shriners Hospital for Children, McGill University, Montreal, QC, Canada., Ward LM; Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada. Lward@cheo.on.ca.; The Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON, K1H 8L1, Canada. Lward@cheo.on.ca.

Publikováno v: Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA [Osteoporos Int] 2023 Jan; Vol. 34 (1), pp. 147-160. Date of Electronic Publication: 2022 Nov 07.

Optimization of DOTAP/chol Cationic Lipid Nanoparticles for mRNA, pDNA, and Oligonucleotide Delivery.

Autor: Sun M; Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Johnson City, NY, 13790, USA.; Department of Biomedical Engineering, The Thomas J. Watson College of Engineering and Applied Science, Binghamton University, Binghamton, NY, 13902, USA., Dang UJ; Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Johnson City, NY, 13790, USA.; Department of Health Sciences, Carleton University, Ottawa, Canada., Yuan Y; Department of Biomedical Engineering, The Thomas J. Watson College of Engineering and Applied Science, Binghamton University, Binghamton, NY, 13902, USA., Psaras AM; Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Johnson City, NY, 13790, USA., Osipitan O; Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Johnson City, NY, 13790, USA.; Department of Biomedical Engineering, The Thomas J. Watson College of Engineering and Applied Science, Binghamton University, Binghamton, NY, 13902, USA., Brooks TA; Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Johnson City, NY, 13790, USA., Lu F; Department of Biomedical Engineering, The Thomas J. Watson College of Engineering and Applied Science, Binghamton University, Binghamton, NY, 13902, USA., Di Pasqua AJ; Department of Pharmaceutical Sciences, School of Pharmacy and Pharmaceutical Sciences, Binghamton University, Johnson City, NY, 13790, USA. dipasqua@binghamton.edu.; Department of Biomedical Engineering, The Thomas J. Watson College of Engineering and Applied Science, Binghamton University, Binghamton, NY, 13902, USA. dipasqua@binghamton.edu.

Publikováno v: AAPS PharmSciTech [AAPS PharmSciTech] 2022 May 09; Vol. 23 (5), pp. 135. Date of Electronic Publication: 2022 May 09.