Bidridistrogene xeboparvovec (SRP-9003) demonstrated a favorable safety profile over 4-5 years in a phase 1/2 trial for limb-girdle muscular dystrophy 2E/R4, with most treatment-related adverse events occurring within 90 days post-infusion.
Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients.
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