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

The life-limiting complications of Duchenne muscular dystrophy (DMD) include loss of lung
function and progressive cardiomyopathy; when patients are treated with assisted …

Objective To describe the quantification of novel dystrophin production in patients with
Duchenne muscular dystrophy (DMD) after long-term treatment with eteplirsen. Methods …

Objective: To correlate time to loss of ambulation (LoA) and different truncating DMD gene
mutations in a large, prospective natural history study of Duchenne muscular dystrophy …

Antisense oligonucleotide (AON)‐mediated exon skipping is an emerging therapeutic for
individuals with Duchenne muscular dystrophy (DMD). Skipping of exons adjacent to …

Patients with Duchenne muscular dystrophy (DMD) show clinically relevant phenotypic
variability, despite sharing the same primary biochemical defect (dystrophin deficiency) …

In-frame exon deletions of the Duchenne muscular dystrophy (DMD) gene produce
internally truncated proteins that typically lead to Becker muscular dystrophy (BMD), a milder …

Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disorder generally caused by
out-of-frame mutations in the DMD gene. In contrast, in-frame mutations usually give rise to …

Objective Respiratory insufficiency is a major complication of Duchenne muscular dystrophy
(DMD). Its progression shows considerable interindividual variability, which has been less …

Background Duchenne muscular dystrophy (DMD) is the most common genetic muscle
disease in human. We aimed to describe the genotype distribution in a large cohort of …