D.4 Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy (EMBARK): Pivotal Phase 3 primary results

Autor: Mendell, JR, Muntoni, F, McDonald, CM, Mercuri, EM, Ciafaloni, E, Komaki, H, Leon-Astudillo, C, Nascimento, A, Proud, C, Schara-Schmidt, U, Veerapandiyan, A, Zaidman, CM, Guridi, M, Murphy, AP, Reid, C, Wandel, C, Darton, E, Mason, S, Potter, RA, Singh, T, Zhang, W, Fontoura, P, Elkins, JS, Rodino-Klapac, LR

Publikováno v: The Canadian Journal of Neurological Sciences; June 2024, Vol. 51 Issue: Supplement 1 pS9-S10, 2p

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

Autor: Rodino-Klapac LR, Lee J, Mulligan RC, Clark KR, Mendell JR

Publikováno v: Neurology; 7/22/2008, Vol. 71 Issue 4, p240-247, 8p

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.

Autor: Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA. JMendell@Sarepta.com.; The Ohio State University, Columbus, OH, USA. JMendell@Sarepta.com.; Sarepta Therapeutics, Inc., Cambridge, MA, USA. JMendell@Sarepta.com., Muntoni F; Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health and Institute of Neurology, University College London and Great Ormond Street Hospital Trust, London, UK., McDonald CM; UC Davis Health, Sacramento, CA, USA., Mercuri EM; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy., Ciafaloni E; University of Rochester Medical Center, Rochester, NY, USA., Komaki H; Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan., Leon-Astudillo C; Department of Pediatrics, University of Florida, Gainesville, FL, USA., Nascimento A; Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Déu, Fundacion Sant Joan de Déu, CIBERER - ISC III, Barcelona, Spain., Proud C; Children's Hospital of the King's Daughters, Norfolk, VA, USA., Schara-Schmidt U; Department of Pediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, University Clinic Essen, University of Duisburg-Essen, Essen, Germany., Veerapandiyan A; Department of Pediatrics, Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR, USA., Zaidman CM; Department of Neurology, Washington University in St. Louis, St. Louis, MO, USA., Guridi M; F. Hoffmann-La Roche, Ltd., Basel, Switzerland., Murphy AP; Roche Products, Ltd., Welwyn Garden City, UK., Reid C; Roche Products, Ltd., Welwyn Garden City, UK., Wandel C; F. Hoffmann-La Roche, Ltd., Basel, Switzerland., Asher DR; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Darton E; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Mason S; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Potter RA; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Singh T; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Zhang W; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Fontoura P; F. Hoffmann-La Roche, Ltd., Basel, Switzerland., Elkins JS; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Rodino-Klapac LR; Sarepta Therapeutics, Inc., Cambridge, MA, USA.

Publikováno v: Nature medicine [Nat Med] 2024 Oct 09. Date of Electronic Publication: 2024 Oct 09.

Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results.

Autor: Mendell JR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA.; Department of Neurology, The Ohio State University, Columbus, OH, USA., Pozsgai ER; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Lewis S; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Griffin DA; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Lowes LP; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Alfano LN; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University, Columbus, OH, USA., Lehman KJ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Church K; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Reash NF; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Iammarino MA; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Sabo B; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Potter R; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Neuhaus S; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Li X; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Stevenson H; Sarepta Therapeutics, Inc., Cambridge, MA, USA., Rodino-Klapac LR; Sarepta Therapeutics, Inc., Cambridge, MA, USA. LRodinoKlapac@Sarepta.com.

Publikováno v: Nature medicine [Nat Med] 2024 Jan; Vol. 30 (1), pp. 199-206. Date of Electronic Publication: 2024 Jan 04.