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Eight week exposure to a high sugar high fat diet results in adiposity gain and alterations in metabolic biomarkers in baboons (Papio hamadryas sp.)

Autor: Tejero M Elizabeth, Voruganti V Saroja, Proffitt J Michael, Garcia-Forey Maggie, Lopez-Alvarenga Juan, Bastarrachea Raul A, Higgins Paul B, Mattern Vicki, Haack Karin, Shade Robert E, Cole Shelley A, Comuzzie Anthony G

Publikováno v: Cardiovascular Diabetology, Vol 9, Iss 1, p 71 (2010)

Abstract Background Baboons (Papio hamadryas Sp.) develop features of the cardiometabolic syndrome and represent a clinically-relevant animal model in which to study the aetiology of the disorder. To further evaluate the baboon as a model for the stu

Externí odkaz: https://doaj.org/article/dd9b4c65801148ccac4a2041b1485b95

Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials.

Autor: Díaz-Manera J; John Walton Muscular Dystrophy Research Centre, Newcastle University International Centre for Life Newcastle upon Tyne, UK. Electronic address: Jordi.Diaz-Manera@newcastle.ac.uk., Hughes D; Lysosomal Storage Disorders Unit, Royal Free Hospital, London, UK., Erdem-Özdamar S; Department of Neurology, Hacettepe University Faculty of Medicine, Ankara, Turkey., Tard C; CHU de Lille, Centre de Référence des Maladies Neuromusculaires Nord Est Ile de France, Lille, France., Béhin A; AP-HP, Centre de Référence des Pathologies Neuromusculaires Nord-Est-Ile de France, Service de Neuromyologie, Hôpital de la Pitié-Salpêtrière, Paris, France., Bouhour F; Referral Centre for Neuromuscular Diseases, Hôpital Neurologique, Lyon-Bron, France., Davison J; Great Ormond Street Hospital NHS Foundation Trust, London, UK; National Institute of Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Hahn SH; Department of Pediatrics, University of Washington School of Medicine, Seattle Children's Hospital, Seattle, WA, USA., Haack KA; Sanofi, Chilly-Mazarin, France., Huynh-Ba O; Sanofi, Chilly-Mazarin, France., Periquet M; Sanofi, Amsterdam, the Netherlands., Tammireddy S; Sanofi, Cambridge, MA, USA., Thibault N; Sanofi, Cambridge, MA, USA., Zhou T; Sanofi, Cambridge, MA, USA., van der Ploeg AT; Center for Lysosomal and Metabolic Diseases, Erasmus MC, University Medical Center, Rotterdam, the Netherlands.

Publikováno v: Molecular genetics and metabolism [Mol Genet Metab] 2024 Dec; Vol. 143 (4), pp. 108608. Date of Electronic Publication: 2024 Nov 08.

Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease.

Autor: Tiraboschi G; Pharmacokinetics Dynamics and Metabolism, Translational Medecine & Early Development, Sanofi R&D, 371 Rue du Pr Blayac, Montpellier, 34184, France. gilles.tiraboschi@sanofi.com., Marchionni D; Pharmacokinetics Dynamics and Metabolism, Translational Medecine & Early Development, Sanofi R&D, 371 Rue du Pr Blayac, Montpellier, 34184, France., Tuffal G; Pharmacokinetics Dynamics and Metabolism, Translational Medecine & Early Development, Sanofi R&D, 371 Rue du Pr Blayac, Montpellier, 34184, France., Fabre D; Pharmacokinetics Dynamics and Metabolism, Translational Medecine & Early Development, Sanofi R&D, 371 Rue du Pr Blayac, Montpellier, 34184, France., Martinez JM; Pharmacokinetics Dynamics and Metabolism, Translational Medecine & Early Development, Sanofi R&D, 371 Rue du Pr Blayac, Montpellier, 34184, France., Haack KA; Sanofi Chilly-Mazarin, 1 Avenue Pierre Brossolette, Chilly-Mazarin, 91385, France., Miossec P; Sanofi Chilly-Mazarin, 1 Avenue Pierre Brossolette, Chilly-Mazarin, 91385, France., Kittner B; Global Pharmacovigilance, Sanofi, Bridgewater, NJ, 08876, USA., Daba N; Global Medical Affairs, Sanofi Gulf Level 3, One JLT, Jumeirah Lake Towers, PO Box 53899, Dubai, UAE., Hurbin F; Pharmacokinetics Dynamics and Metabolism, Translational Medecine & Early Development, Sanofi R&D, 371 Rue du Pr Blayac, Montpellier, 34184, France.

Publikováno v: Journal of pharmacokinetics and pharmacodynamics [J Pharmacokinet Pharmacodyn] 2023 Dec; Vol. 50 (6), pp. 461-474. Date of Electronic Publication: 2023 Aug 03.

Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.

Autor: Dimachkie MM; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany. mdimachkie@kumc.edu., Barohn RJ; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Byrne B; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Goker-Alpan O; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Kishnani PS; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Ladha S; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Laforêt P; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Mengel KE; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Peña LDM; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Sacconi S; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Straub V; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Trivedi J; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Van Damme P; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., van der Ploeg AT; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Vissing J; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Young P; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Haack KA; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Foster M; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Gilbert JM; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Miossec P; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Vitse O; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Zhou T; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Schoser B; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany.

Publikováno v: Neurology [Neurology] 2022 Aug 01; Vol. 99 (5), pp. e536-e548. Date of Electronic Publication: 2022 Aug 01.