DMD Boys Experience Biological, Functional Gains in SRP-9001 Study

DMD Boys Experience Biological, Functional Gains in SRP-9001 Study

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DMD Boys Experience Biological, Functional Gains in SRP-9001 Study

The investigational gene therapy SRP-9001 safely leads to the production of a functional form of dystrophin in boys with Duchenne muscular dystrophy (DMD) — and to improvements in their motor abilities, according to top-line results of a Phase 2 trial. However, following almost one year of the Sarepta Therapeutics treatment, functional improvements relative to a placebo were only statistically significant in younger boys ages 4 to 5, as reported in a press release. Study 102 (NCT03769116)…

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