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

Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused
by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of …

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

Although genetic and cell-mediated approaches are currently showing genuine promise in
preclinical and clinical trials, there remains considerable interest in the development of …

Introduction: Since the identification of the dystrophin gene in 1986, a cure for Duchenne
muscular dystrophy (DMD) has yet to be discovered. Presently, there are a number of …

Background To explore clinical heterogeneity of Duchenne muscular dystrophy (DMD),
viewed as a major obstacle to the interpretation of therapeutic trials Methodology/Principal …

Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of
functional dystrophin protein. Patients experience progressive muscle weakness …

Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Genetic modifiers can serve as biomarkers for outcomes in DMD. Modifiers can alter
strength and ambulation in muscular dystrophy, and these same features can be used as …

Introduction Duchenne muscular dystrophy (DMD) is the result of X-chromosome-linked
mutations to the dystrophin protein gene that prevent the normal development and repair of …