Clinical Trial Update: A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency) [Phase 1]

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Summary

Phase 1 gene transfer trial SRP-9003 for limb-girdle muscular dystrophy type 2E/beta-sarcoglycan deficiency is active but not recruiting, evaluating safety, tol…

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Diseases / indications

• Limb-girdle Muscular Dystrophy Type 2e

Drugs mentioned

• SRP-9003

Snippet

Sponsor: Sarepta Therapeutics, Inc. | Conditions: Limb Girdle Muscular Dystrophy | Phase 1 | Status: ACTIVE_NOT_RECRUITING