The timely and accurate genetic diagnosis of Duchenne muscular dystrophy (DMD) enables
prompt initiation of disease management and genetic counseling and optimal patient care …

Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …

Background: Publication of comprehensive clinical care guidelines for Duchenne muscular
dystrophy (DMD) in 2010 was a milestone for DMD patient management. Our CARE-NMD …

Importance Corticosteroids improve strength and function in boys with Duchenne muscular
dystrophy. However, there is uncertainty regarding the optimum regimen and dosage …

Dystrophinopathies are a group of X-linked neuromuscular disorders arising from mutations
in the dystrophin (DMD) gene. The DMD gene encodes dystrophin protein, which plays an …

Background: In the absence of a curative treatment for Duchenne Muscular Dystrophy
(DMD), corticosteroid therapy (prednisone, deflazacort) has been adopted as the standard of …

Background Currently, there is no cure for Duchenne and Becker muscular dystrophies
(DMD/BMD). However, clinical trials with new therapeutic strategies are being conducted or …

Abstract Background Female carriers of Duchenne muscular dystrophy (DMD), although
usually asymptomatic, develop muscle weakness up to 17% of the time, and a third present …

In 2005, the American Academy of Neurology and the Child Neurology Society published a
practice parameter, based primarily on studies that involved 6 to 18 months of treatment …

Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy
affecting~ 1: 5000 live male births. Following the identification of pathogenic variations in the …