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Duchenne Muscular Dystrophy

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

FDA Panel Rejects Deramiocel Cardiac Claim After Lancet Publishes HOPE‑3 Results

A 9–3 advisory vote and critical FDA reviews increase doubt about final approval ahead of the August 22 PDUFA date.

HHS Adds Duchenne Muscular Dystrophy and MLD to Newborn Screening Recommendations

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