Health ❯ Healthcare ❯ Rare Diseases

Duchenne Muscular Dystrophy

Treatment Options Therapeutic Development Cell Therapy Orphan Drug Designation Diagnosis and Treatment Metachromatic Leukodystrophy

HHS Adds Duchenne Muscular Dystrophy and MLD to Newborn Screening Recommendations

States will decide on adoption, with health officials urging early screening to enable time-sensitive treatment.

Capricor’s HOPE-3 Trial Reports Cardiac and Muscle Gains in Duchenne Muscular Dystrophy

FDA Opens Door to Reconsider Capricor’s Duchenne Cell Therapy Based on HOPE-3 Data

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