DMD CLINICAL THERAPIES II

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Duchenne muscular dystrophy (DMD) patients experience progressive degeneration of skeletal muscles including those involved in respiration. Well characterized respiratory decline in glucocorticoid-treated DMD patients includes forced vital capacity % predicted (FVC%p) decline ≥5% per year between the ages of 10 and 18 years. We evaluated eteplirsen treatment effect on FVC%p annual change in three eteplirsen clinical trials compared to well-matched natural history controls from the Cooperative International Neuromuscular Research Group (CINRG).

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