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)

Study Summary

The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.

Conditions

Limb Girdle Muscular Dystrophy

Interventions / Drugs

SRP-9003

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment6 participants

Primary Completion—

Lead SponsorSarepta Therapeutics, Inc.

Data Retrieved2026-04-11 02:55:56.327639+00:00

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