Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due
to mutations in the dystrophin gene. It is characterized by progressive muscle weakness and …

Many neuromuscular diseases are genetically inherited or caused by mutations in motor
function proteins. Two of the most prevalent neuromuscular diseases are Duchenne …

Intra‐arterial transplantation of mesoangioblasts proved safe and partially efficacious in
preclinical models of muscular dystrophy. We now report the first‐in‐human, exploratory …

Various therapeutic approaches have been studied for the treatment of Duchenne muscular
dystrophy (DMD), but none of these approaches have led to significant long-term effects in …

For many neuromuscular disorders, including Duchenne muscular dystrophy, spinal
muscular atrophy and myotonic dystrophy, the genetic causes are well known. Gene therapy …

Background and purpose: Duchenne muscular dystrophy (DMD) is a lethal progressive
pediatric muscle disorder and genetically inherited as an X-linked disease that caused by …

Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by
progressive muscle degeneration. Mutations in the DMD gene result in the absence of …

Duchenne muscular dystrophy (DMD) is a X-linked, progressive childhood myopathy
caused by mutations in the dystrophin gene, one of the largest genes in the genome. It is …

The purpose of this study was to explore the use of iterative decomposition of water and fat
with echo asymmetry and least-squares estimation Carr–Purcell–Meiboom–Gill (IDEAL …

Background/aims: Pediatric rare disease presents a challenging situation of high unmet
need and a limited pool of potential clinical trial participants. Understanding perspectives of …