Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in
2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved …

Abstract Background Duchenne Muscular Dystrophy (DMD) is a rapidly progressive, lethal
neuromuscular disorder, present from birth, which occurs almost exclusively in males. We …

Background Duchenne muscular dystrophy (DMD) is a severe, progressive, and rare
neuromuscular, X-linked recessive disease. Dystrophin deficiency is the underlying cause of …

Objective To continue evaluation of the long‐term efficacy and safety of eteplirsen, a
phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with …

Background Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy
of childhood. Untreated, this incurable disease, which has an X‐linked recessive …

Muscular dystrophies are primary diseases of muscle due to mutations in more than 40
genes, which result in dystrophic changes on muscle biopsy. Now that most of the genes …

Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …

Importance Vamorolone is a synthetic steroidal drug with potent anti-inflammatory
properties. Initial open-label, multiple ascending dose-finding studies of vamorolone among …

Introduction: The development of adeno-associated virus (AAV) vectors as safe vehicles for
in vivo delivery of therapeutic genes has been a major milestone in the advancement of …

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 …