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Update on the Treatment of Duchenne Muscular Dystrophy

  • Nerve and Muscle (M Hirano and LH Weimer, Section Editors)
  • Published:
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Abstract

Duchenne muscular dystrophy is the most severe childhood form of muscular dystrophy caused by mutations in the gene responsible for dystrophin production. There is no cure, and treatment is limited to glucocorticoids that prolong ambulation and drugs to treat the cardiomyopathy. Multiple treatment strategies are under investigation and have shown promise for Duchenne muscular dystrophy. Use of molecular-based therapies that replace or correct the missing or nonfunctional dystrophin protein has gained momentum. These strategies include gene replacement with adeno-associated virus, exon skipping with antisense oligonucleotides, and mutation suppression with compounds that “read through” stop codon mutations. Other strategies include cell therapy and surrogate gene products to compensate for the loss of dystrophin. All of these approaches are discussed in this review, with particular emphasis on the most recent advances made in each therapeutic discipline. The advantages of each approach and challenges in translation are outlined in detail. Individually or in combination, all of these therapeutic strategies hold great promise for treatment of this devastating childhood disease.

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Disclosure

L.R. Rodino-Klapac: none; J.R. Mendell: principal investigator in the eteplirsen trial, for which he received no personal compensation; Z. Sahenk: none.

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Authors and Affiliations

  1. Department of Pediatrics, The Ohio State University, and Nationwide Children’s Hospital, Columbus, OH, 43210, USA

    Louise R. Rodino-Klapac, Jerry R. Mendell & Zarife Sahenk

  2. Department of Neurology, The Ohio State University, and Nationwide Children’s Hospital, Columbus, OH, 43210, USA

    Jerry R. Mendell & Zarife Sahenk

  3. Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, 700 Children’s Dr., Room WA3011, Columbus, OH, 43205, USA

    Louise R. Rodino-Klapac, Jerry R. Mendell & Zarife Sahenk

Authors
  1. Louise R. Rodino-Klapac
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  2. Jerry R. Mendell
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  3. Zarife Sahenk
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Corresponding authors

Correspondence to Louise R. Rodino-Klapac or Zarife Sahenk.

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This article is part of the Topical Collection on Nerve and Muscle

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Rodino-Klapac, L.R., Mendell, J.R. & Sahenk, Z. Update on the Treatment of Duchenne Muscular Dystrophy. Curr Neurol Neurosci Rep 13, 332 (2013). https://doi.org/10.1007/s11910-012-0332-1

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