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.
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.
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Spotlight
The Complexities of Gene Therapy Manufacturing
Learn more about what goes into manufacturing gene therapies and Sarepta’s approach to meeting the challenges inherent in making these transformative therapies a reality for patients with rare diseases.
Highlights
Our FDA-Approved Treatments
Lean more about our FDA-approved treatments.
A Day in The Life at Sarepta
Hear from employees and how our mission drives their work.