Updated results from several trials of SRP-9001 (delandistrogene moxeparvovec) revealed a wealth of positives, including functional benefits in patients with the muscle-wasting disease after one to fours years of follow-up, pointing to a durable effect of the gene therapy.
Sarepta’s gene therapy – like rivals from Pfizer and Solid Biosciences – codes for a shortened form of the dystrophin protein that is deficient in patients with the muscle-wasting disease.
