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Chugai Launches ELEVIDYS as Japan's First Gene Therapy for Duchenne Muscular Dystrophy

Feb 28, 2026

Chugai Pharmaceutical launched ELEVIDYS in Japan as the first regenerative medical product for Duchenne muscular dystrophy following its listing on the National Health Insurance reimbursement price list on February 20, 2026.

PTC Therapeutics Withdraws Translarna NDA After FDA Cites Insufficient Efficacy Data

Feb 13, 2026

PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.