Duchenne muscular dystrophy (DMD) is a fatal genetic disorder caused by mutations in the DMD gene. Here, the authors develop MyoAAV-UA, a compact utrophin activator, as a promising universal ...
"We remain dedicated to building broader awareness of the devastating impact of rare, progressive neuromuscular disorders including DMD, DM1 and FSHD as well as rare cardiomyopathies and to ...
Aside from muscular dystrophy, other examples of rare diseases include hemophilia, an inherited blood disorder that causes excessive bleeding and bruising; phenylketonuria, an inherited disorder ...
The research topic “New Therapeutic Target and Drug Discovery for Neurological Disorders, Volume II” encompasses 32 articles contributed by 236 authors. This comprehensive collection presents a wide ...
The topics we cover include: neurophysiology of muscle contraction neuromuscular transmission central nervous system control of peripheral processes immunology of inflammatory muscle nerve disorders ...
The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease ... form for patients with spinal muscular atrophy aged 2 years and older ...
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