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.
Press Releases
- Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
- Gene Therapy Pioneer Jerry R. Mendell, M.D., Named to Inaugural TIME100 Health List of the 100 Most Influential People in Global Health
- Sarepta Therapeutics Announces First Quarter 2024 Financial Results and Recent Corporate Developments
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.
RNA-Targeted Therapies: A Development Strategy for Protein Upregulation
Learn more about the biochemical structure of phosphodiamidate morpholino oligomers (PMOs) and PPMOs and their mechanism.
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.
