Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease This is the 2nd orphan drug designation granted to the skeletal muscle-specific ...
They were generously donated by patients with NMD. Samples are routinely collected from patients attending The John Walton Muscular Dystrophy Research Centre clinics, those participating in clinical ...
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Background Fatty atrophy is a combination of reduced muscle size and the replacement of muscle tissue with fat seen in patients with chronic neuromuscular diseases (NMD). Accurate quantification of ...