Active, not recruitingPhase 1NCT05876780

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)

Studying Beta-sarcoglycan-related limb-girdle muscular dystrophy R4

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Key facts

Sponsor
Sarepta Therapeutics, Inc.
Principal Investigator
Medical Director
Sarepta Therapeutics, Inc.
Intervention
SRP-9003(genetic)
Enrollment
6 enrolled
Eligibility
4-50 years · All sexes
Timeline
20222028

Study locations (2)

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT05876780 on ClinicalTrials.gov

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