Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

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 …

Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD
gene mutations. Phenotype is variable with loss of ambulation in late teenage or late mid-life …

Intra‐arterial transplantation of mesoangioblasts proved safe and partially efficacious in
preclinical models of muscular dystrophy. We now report the first‐in‐human, exploratory …

Duchenne muscular dystrophy (DMD) is an X-linked disease caused by mutations in the
DMD gene and loss of the protein dystrophin. The absence of dystrophin leads to myofiber …

The high number of somatic mutations in the melanoma genome associated with cumulative
ultra violet (UV) exposure has rendered it one of the most difficult of cancers to treat. With …

Duchenne muscular dystrophy (DMD) is a genetic muscle disorder caused by mutations in
the Dmd gene resulting in the loss of the protein dystrophin. Patients do not only experience …

Objective: To conduct a randomized trial to test the primary hypothesis that once-daily
tadalafil, administered orally for 48 weeks, lessens the decline in ambulatory ability in boys …

Objective Duchenne and Becker muscular dystrophies (DBMD) are allelic disorders caused
by mutations in dystrophin. Adults with DBMD develop life‐threatening cardiomyopathy …