What type of study is this?

This is a Phase 3 study (also classified as: RCT).

September 28, 2025

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

Key Points

Treatment with delandistrogene moxeparvovec shows significant improvement in function over two years.
Key safety and efficacy data were reported from a phase 3 randomized controlled trial with a placebo group.
Micro-dystrophin expression contributes to the stabilization of muscle function, impacting DMD progression.
The study underscores the promise of gene therapy as a new approach for managing DMD.

Abstract

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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