Phase 3 trial reveals safety and efficacy of gene therapy in DMD patients, highlighting micro-dystrophin expression.
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, two-part study, assesses delandistrogene moxeparvovec in DMD.
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Seelmann et al. (2025) studied this question.
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