Abstract Background Duchenne Muscular Dystrophy (DMD) is a rapidly progressive, lethal
neuromuscular disorder, present from birth, which occurs almost exclusively in males. We …

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

For many neuromuscular diseases (NMDs), cardiac disease represents a major cause of
morbidity and mortality. The management of cardiac disease in NMDs is made challenging …

Muscular Dystrophy, convened a Working Group meeting on July 2014, in Bethesda, MD, to
explore clinical and research questions related to cardiac disease in patients with DMD. As …

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in
childhood. It is caused by mutations of the DMD gene, leading to progressive muscle …

Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is
an ongoing, multicenter registry providing real-world evidence regarding ataluren use in …

Duchenne muscular dystrophy (DMD) is a genetic, progressive neuromuscular condition
that is marked by the long-term muscle deterioration with significant implications of …

Duchenne muscular dystrophy (DMD) results in a progressive cardiomyopathy that
produces significant morbidity and mortality. To improve the quality of life in patients with …

Duchenne muscular dystrophy (DMD) afflicts 1 in 5000 newborn males, leading to
progressive muscle weakening and the loss of ambulation between the ages of 8 and 12 …

Duchenne muscular dystrophy (DMD), the most severe form of dystrophinopathy, is quite
homogeneous with regards to its causative biochemical defect, ie, complete dystrophin …