Advances in the pharmacotherapeutic management of duchenne muscular dystrophy: an update.

Abstract excerpt

INTRODUCTION: Duchenne muscular dystrophy (DMD) is a severe X-linked disorder caused by mutations in the dystrophin gene, resulting in progressive skeletal muscle degeneration, loss of ambulation, and eventual cardiopulmonary failure. Although survival has improved, DMD remains incurable and associated with substantial morbidity and premature mortality. AREAS COVERED: A comprehensive literature search was...

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