7/28/2025

Update on Shipments of Gene Therapy for Ambulatory Individuals with Duchenne Muscular Dystrophy

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Some see slow and steady scientific progress.

We see a revolution.

Sarepta is a global biotechnology company on an urgent mission: engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases.

We’re ushering in a new era of drug development, with the goal of shortening the time from lab to patient, building the world’s largest gene therapy manufacturing capacity, and rethinking access and reimbursement models for revolutionary new treatments. We are in a daily race to transform genetic understanding into genetic medicine. Because every day is an opportunity to change the lives of people living with rare disease.

Patients can’t wait for the next breakthrough in medical research.

So neither will we.

While there may be as many as 7,000 rare diseases, only a small percentage have treatments. That is why we are relentless in our dedication: Leverage the best science to help as many patients as possible. Today, we are doing just that in Duchenne muscular dystrophy, in six forms of limb-girdle muscular dystrophy, and in Charcot-Marie-Tooth disease, among others. Sarepta will always follow the science and continuously evaluate other diseases and modalities to pursue.