Clinical Trial Update: Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C [Phase 1]

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Summary

Phase 1 clinical trial evaluating AAV1-gamma-sarcoglycan gene therapy for Limb Girdle Muscular Dystrophy Type 2C (gamma-sarcoglycanopathy) has completed.

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Diseases / indications

• Gamma-sarcoglycanopathy
• Limb-girdle Muscular Dystrophy Type 2c

Drugs mentioned

• AAV1-gamma-sarcoglycan gene therapy

Snippet

Sponsor: Genethon | Conditions: Limb Girdle Muscular Dystrophy Type 2C, Gamma-sarcoglycanopathy | Phase 1 | Status: COMPLETED