SRPT Finishes Enrolment in Late-Stage Study for Rare Muscular Disorder

Sarepta Therapeutics (SRPT) announced that it has completed enrolment and dosing in the phase III EMERGENE study evaluating its investigational gene therapy, SRP-9003 (bidridistrogene xeboparvovec) for treating limb-girdle muscular dystrophy (LGMD) Type 2E/R4 (LGMD2E/R4, or beta-sarcoglycanopathy).Data from the EMERGENE study is expected in the first half of 2025. If the data is positive, Sarepta intends to submit a regulatory filing seeking accelerated approval for SRP-9003 in the given indication before t ...