Duchenne muscular dystrophy (DMD) is a debilitating and ultimately lethal disease involving
progressive muscle degeneration and neurological dysfunction. DMD is caused by …

Aims Variable degrees of inflammation, necrosis, regeneration and fibrofatty replacement
are part of the pathological spectrum of the dystrophic process in alpha dystroglycanopathy …

Objective (s): Duchene muscular dystrophy (DMD) is a progressive neuromuscular disease
caused by mutations in the DMD gene, resulting in the absence of dystrophin expression …

Progressive loss of muscle and muscle function is associated with significant fibrosis in
Duchenne muscular dystrophy (DMD) patients. Halofuginone, an analog of febrifugine …

Neuroblastoma is a neural crest cell-derived pediatric tumor characterized by high inter-and
intra-tumor heterogeneity, and by a poor outcome in advanced stages. Patient-derived …

Abstract GlaxoSmithKline and Astex Pharmaceuticals recently disclosed the discovery of the
potent H-PGDS inhibitor GSK2894631A 1a (IC 50= 9.9 nM) as part of a fragment-based drug …

Objective Duchenne muscular dystrophy has been a launching pad for patient-focused drug
development (PFDD). Yet, PFDD efforts have largely neglected non-ambulatory patients. To …

Up to 15% of Duchenne's muscular dystrophy (DMD) is caused by nonsense mutations (nm-
DMD). In this study, we aimed to evaluate the age at diagnosis, presentations, and …

In recent years, several new treatments have been developed, or are currently tested in
trials, for Duchenne muscular dystrophy (DMD), a rare, X-linked recessive, severely …

Duchenne muscular dystrophy (DMD) causes severe disability of children and death of
young men, with an incidence of approximately 1/5,000 male births. Symptoms appear in …