NCT01344798

Phase I Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

Study Summary

The purpose of this trial is to study the evaluation of clinical safety and feasibility of gene therapy in patients with limb girdle muscular dystrophy type 2C (gamma-sarcoglycanopathy).

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Interventions

AAV1-gamma-sarcoglycan vector injectionBIOLOGICAL
single intramuscular injection into carpi radialis muscle under open procedure

Study Locations

FacilityCityStateCountry
Hôpital Pitié-SalpêtrièreParisFrance

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026