Cardiopulmonary phenotypic variability and discordance in Duchenne muscular dystrophy: Implications for new therapies.

Abstract excerpt

Neuromuscular respiratory medicine has traditionally focused on assisted lung ventilation and mucus clearance. These therapies have prolonged survival for patients with Duchenne muscular dystrophy (DMD). However, the field is rapidly evolving in a new direction: it is being revolutionized by molecular and genetic therapies. A good correlation between a patient's dystrophin mutation and his cardiopulmonary...

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