C. M. Zaidman

Long-Term Functional Outcomes, Safety, and Micro-Dystrophin Expression Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results

I. Seelmann, Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald et autres

Background/Purpose: Delandistrogene moxeparvovec (rAAVrh74 vector-based gene therapy approved in the United States and other select countries) delivers a transgene encoding micro-dystrophin, an engineered, functional form of dystrophin that stabilizes/slows Duchenne muscular dystrophy (DMD) progression. EMBARK (NCT05096221), a Phase 3, randomized, double-blind, placebo-controlled, …

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21OAssessment of cardiac outcomes in delandistrogene moxeparvovec clinical trials for Duchenne muscular dystrophy

A. Veerapandiyan, Jane Bourke, John Day, CM McDonald et autres

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Safety and Efficacy of Delandistrogene Moxeparvovec versus Placebo in Duchenne Muscular Dystrophy (EMBARK): Pivotal Phase 3 Primary Results

I. Seelmann, Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald et autres

Background/Purpose: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations. Delandistrogene moxeparvovec is an adeno-associated viral vector-based gene transfer therapy, designed to compensate for absent functional dystrophin in DMD by delivering a transgene encoding engineered micro-dystrophin, which retains key functional wild-type …

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The Epidys Givinostat Study in DMD: Supportive Results

Eugenio Maria Mercuri, Claudia Brogna, C. M. Zaidman, W. Müller-Felber et autres

Background/Purpose: The Phase 3 Epidys study with givinostat in DMD met its primary endpoint with consistent results in the key secondary endpoints. The primary analysis was conducted in boys with baseline MRS vastus lateralis fat fraction >5 to 30% (target population, N …

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P.128 Integrated analyses of data from clinical trials of delandistrogene moxeparvovec in DMD

C. M. Zaidman, P. Shieh, C. Proud, C. McDonald et autres

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P.129 One-year data from ENDEAVOR, a phase 1b trial of delandistrogene moxeparvovec in boys with DMD

C. M. Zaidman, Crystal M. Proud, CM McDonald, Stefanie Mason et autres

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