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Press Releases

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  • 06/15/2025
    Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne
  • 06/04/2025
    U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4
  • 05/21/2025
    Sarepta Provides Update on UK Dosing in ENVISION Study of ELEVIDYS for the treatment of Duchenne Muscular Dystrophy

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  • Video file
    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.

    Learn more
  • Video file
    Some think a cure for a rare disease is a miracle.

    Our scientists would disagree.

    Sarepta is engineering solutions for rare diseases with science that is on the forefront of precision genetic medicine. Our platforms include: gene therapy, RNA technologies, and gene editing. We are in a daily race to save lives stolen or impacted by rare disease.

    Learn more
  • Video file
    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.

    Learn more
  • Join Us
    The opportunity to transform lives is breathtaking.

    Join us.

    We see endless possibilities for changing the future of medicine. We’re looking for people who see unlimited potential in themselves, opportunity where others see roadblocks, and solutions limited only by the power of imagination and the drive to do things differently.

    Learn About Working at Sarepta

Exploring the Potential of Gene Therapies for Limb-girdle Muscular Dystrophies

Dr. Louise Rodino-Klapac discusses the science of limb-girdle muscular dystrophies (LGMDs) and how our gene therapy engine will help engineer potential therapies for this group of inherited neuromuscular diseases.

Karen

Sarepta's gene therapy engine at work in LGMD2E research

Sarepta’s gene therapy engine provides a framework for creating a steady stream of new therapies for devastating diseases, such as limb-girdle muscular dystrophies.

Learn more about our LGMD research

Press Releases

See All
  • 06/15/2025
    Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne
  • 06/04/2025
    U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4
  • 05/21/2025
    Sarepta Provides Update on UK Dosing in ENVISION Study of ELEVIDYS for the treatment of Duchenne Muscular Dystrophy

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The Complexities of Gene Therapy Manufacturing

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