Duchenne muscular dystrophy (DMD) is a disease linked to the X-chromosome which
affects 1 in 3,600-6,000 newborn males. It is manifested by the absence of the dystrophin …

Muscular dystrophies are a clinically and heterogeneous group of disorders that all share
clinical characteristics of progressive muscular weakness. Duchenne muscular dystrophy …

Background Duchenne muscular dystrophy (DMD) is an inherited neuromuscular disorder of
childhood with a devastating disease course. Several targeted gene therapies and …

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 …

Duchenne muscular dystrophy (DMD) is the commonest inherited neuromuscular disorder of
childhood and mainly affects males. Over the course of the last century, the average life …

A recent Cochrane review showed evidence that corticosteroids are effective in Duchenne
muscular dystrophy (DMD) patients to improve muscle strength and to extend the ambulant …

The past 18 months have seen a strong increase in the number of exciting reports on novel
therapeutic agents for DMD. Exon-skipping agents have been fine-tuned to improve tissue …

The pharmacological stimulation of AMP-activated protein kinase (AMPK) via metabolic
enhancers has been proposed as potential therapeutic strategy for Duchenne muscular …

Background and Objectives Clinical trials of genotype-targeted treatments in Duchenne
muscular dystrophy (DMD) traditionally compare treated patients with untreated patients with …

Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of
childhood for which there is no cure. This authoritative guide provides a clear overview of …