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limb-girdle muscular dystrophy type R5 (previously named LGMD2C) research hub

1 indexed studies · 0 currently recruiting

authorisedPhase I and Phase II (Integrated)- First administration to humans
A Phase I/II, open-label, dose escalation study to evaluate the safety of 2 doses of intravenous ATA-200, an adeno-associated viral vector carrying the human gamma-sarcoglycan gene, in patients with gamma-sarcoglycanopathy (limb-girdle muscular dystrophy LGMDR5, formerly LGMD2C).
France · Italy