Background: Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy
(DMD) have indicated greater disease variability in terms of progression than expected. In …

Duchenne muscular dystrophy (DMD) is a severe genetic disease with loss of walking at a
mean age of 9.5 years and death in late teens or twenties. Despite major advances in …

Duchenne muscular dystrophy (DMD) is the commonest and best-known of the muscular
dystrophies. Being an X-linked disorder, it affects mainly boys. The disease gene was …

In June 2014 TREAT-NMD organised a workshop at The Wellcome Trust head office in
London to discuss issues such as natural history, outcome measures, efficacy …

ABSTRACT Introduction: In Duchenne muscular dystrophy (DMD), fat replacement of muscle
may be a useful endpoint in trials of therapy, although progression in different muscle …

Duchenne muscular dystrophy (DMD), an inherited X-linked recessive disorder, is
characterized by progressive symmetric muscle weakness and gait disturbance, with onset …

An ENMC workshop took place in Naarden on 23-25 January 2014 and involved 27
participants from eight countries, and included clinicians, researchers, drug company and …

Muscular dystrophies are a clinically and genetically heterogeneous group of skeletal
muscle-wasting diseases. Even for experts in the field of neuromuscular diseases, it is …

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 …

Introduction: Evidence-based therapeutics in Duchenne muscular dystrophy (DMD) has
been limited to corticosteroids for the past 30 years. There have been a host of other …