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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AAV1-gamma-sarcoglycan vector injectionBIOLOGICAL
single intramuscular injection into carpi radialis muscle under open procedure
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Hôpital Pitié-Salpêtrière | Paris | France |