Recent developments in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy

Wendy K.M. Liew, Peter B. Kang

Research output: Contribution to journalReview articlepeer-review

Abstract

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.

Original languageEnglish (US)
Pages (from-to)147-160
Number of pages14
JournalTherapeutic Advances in Neurological Disorders
Volume6
Issue number3
DOIs
StatePublished - May 2013
Externally publishedYes

Bibliographical note

Funding 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.

Keywords

  • Duchenne muscular dystrophy
  • spinal muscular atrophy
  • therapy

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