Therapeutic advances in muscular dystrophy.

Doris G. Leung, Kathryn Rae Wagner

Research output: Contribution to journalArticle

Abstract

The muscular dystrophies comprise a heterogeneous group of genetic disorders that produce progressive skeletal muscle weakness and wasting. There has been rapid growth and change in our understanding of these disorders in recent years, and advances in basic science are being translated into increasing numbers of clinical trials. This review will discuss therapeutic developments in 3 of the most common forms of muscular dystrophy: Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and myotonic dystrophy. Each of these disorders represents a different class of genetic disease (monogenic, epigenetic, and repeat expansion disorders), and the approach to therapy addresses the diverse and complex molecular mechanisms involved in these diseases. The large number of novel pharmacologic agents in development with good biologic rationale and strong proof of concept suggests there will be an improved quality of life for individuals with muscular dystrophy.

Original languageEnglish (US)
Pages (from-to)404-411
Number of pages8
JournalAnnals of Neurology
Volume74
Issue number3
StatePublished - Sep 2013
Externally publishedYes

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Muscular Dystrophies
Inborn Genetic Diseases
Facioscapulohumeral Muscular Dystrophy
Myotonic Dystrophy
Duchenne Muscular Dystrophy
Muscle Weakness
Epigenomics
Skeletal Muscle
Therapeutics
Quality of Life
Clinical Trials
Growth

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology
  • Medicine(all)

Cite this

Therapeutic advances in muscular dystrophy. / Leung, Doris G.; Wagner, Kathryn Rae.

In: Annals of Neurology, Vol. 74, No. 3, 09.2013, p. 404-411.

Research output: Contribution to journalArticle

Leung, DG & Wagner, KR 2013, 'Therapeutic advances in muscular dystrophy.', Annals of Neurology, vol. 74, no. 3, pp. 404-411.
Leung, Doris G. ; Wagner, Kathryn Rae. / Therapeutic advances in muscular dystrophy. In: Annals of Neurology. 2013 ; Vol. 74, No. 3. pp. 404-411.
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