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Duchenne Muscular Dystrophy

Sarepta Shares Plunge After ESSENCE Phase 3 Miss in Duchenne Trial

The company will seek FDA guidance on converting its exon-skipping drugs from accelerated to full approval.

Regulators Halt Elevidys Gene Therapy Worldwide After Safety Failures

Sarepta Defies FDA, Keeps Duchenne Gene Therapy on the Market

Sarepta Halts Elevidys Shipments for Non-Ambulatory Patients After Second Fatal Liver Failure