Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. After discussions with the FDA, the biotech has launched a ...
This study reveals abnormal iron accumulation in the muscles of patients with Facioscapulohumeral Muscular Dystrophy (FSHD) and in mice expressing muscle-specific DUX4 (DUX4-Tg mice). Contrary to ...
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