Pediatric neuromuscular disorders comprise a large variety of disorders that can be classified based on their neuroanatomical localization, patterns of weakness, and laboratory test results. Over the last decade, the field of translational research has been active with many ongoing clinical trials. This is particularly so in two common pediatric neuromuscular disorders: Duchenne muscular dystrophy and spinal muscular atrophy. Although no definitive therapy has yet been found, numerous active areas of research raise the potential for novel therapies in these two disorders, offering hope for improved quality of life and life expectancy for affected individuals.
Bibliographical noteFunding Information:
WKML has received funding for travel from ISIS Pharmaceuticals. PBK has received travel funding, research support, and an honorarium from ISIS Pharmaceuticals, as well as travel funding from PTC Therapeutics.
- Duchenne muscular dystrophy
- spinal muscular atrophy