March 27th 2023
The company's persistence in bringing NurOwn to the market amidst negative regulatory feedback has garnered an AdComm meeting.
Sarepta’s SRP-9001 Shows Functional Improvements in DMD Integrated Analysis
March 22nd 2023Presented at MDA 2023, the gene therapy (also known as delandistrogene moxeparvovec) showed significant improvements on NSAA total score, 10-meter walk/run scores, and time to rise scores relative to an external control cohort.
FDA Revisits Need for Advisory Committee on Sarepta’s DMD Gene Therapy
March 18th 2023Ahead of the May 29, 2023, action date, the FDA is now seeking to hold an advisory committee meeting on Sarepta’s investigational agent SRP-9001 (also known as delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy.
Microdystrophin Proposed as a Surrogate End Point in DMD
March 16th 2023Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, discussed the potential use of microdystrophin as an end point for clinical evaluations of gene therapies for DMD.