DMD Gene Therapy, SRP-9001, Showing ‘Robust’ Micro-dystrophin in Trial

DMD Gene Therapy, SRP-9001, Showing ‘Robust’ Micro-dystrophin in Trial

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DMD Gene Therapy, SRP-9001, Showing ‘Robust’ Micro-dystrophin in Trial

Sarepta Therapeutics‘ investigational gene therapy SRP-9001 showed early signs of biologic efficacy in muscle biopsies from 11 boys with Duchenne muscular dystrophy (DMD) treated in a Phase 1 clinical trial, the company announced in a press release. “Robust” expression of micro-dystrophin was evident in cells at 12 weeks post-treatment, and no new safety signals were reported. “We are delighted by these seminal results … our first trial results with SRP-9001 made by our commercial-scale manufacturing process,”…

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