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Sarepta Therapeutics Reports Sustained Functional Improvement Two Years After Treatment With SRP-9001, Its Investigational Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy


Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced two-year follow up results from four Duchenne muscular dystrophy (DMD) clinical trial participants who received SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of micro-dystrophin protein. Results presented at the 25th International Annual Congress of the World Muscle Society demonstrated that two years after a one-time infusion of SRP-9001, trial participants exhibited a mean 7.0 point improvement on the North Star Ambulatory Assessment (NSAA) compared to baseline.

“We continue to be encouraged by the safety profile and enduring treatment response that we have seen to date with SRP-9001 gene transfer therapy,” said Doug Ingram, President and CEO, Sarepta.

Sarepta Therapeutics Reports Sustained Functional Improvement Two Years After Treatment With SRP-9001, Its Investigational Micro-Dystrophin Gene Therapy For Duchenne Muscular Dystrophy