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Molecular Diagnosis and Novel Therapies for Neuromuscular Diseases

Authors
  • Maruyama, Rika
  • Yokota, Toshifumi1
  • 1 The Friends of Garrett Cumming Research & Muscular Dystrophy Canada, HM Toupin Neurological Science Research Chair, Edmonton, AB T6G 2H7, Canada
Type
Published Article
Journal
Journal of Personalized Medicine
Publisher
MDPI
Publication Date
Sep 16, 2020
Volume
10
Issue
3
Identifiers
DOI: 10.3390/jpm10030129
PMID: 32947786
PMCID: PMC7564006
Source
PubMed Central
Keywords
License
Green

Abstract

With the development of novel targeted therapies, including exon skipping/inclusion and gene replacement therapy, the field of neuromuscular diseases has drastically changed in the last several years. Until 2016, there had been no FDA-approved drugs to treat Duchenne muscular dystrophy (DMD), the most common muscular dystrophy. However, several new personalized therapies, including antisense oligonucleotides eteplirsen for DMD exon 51 skipping and golodirsen and viltolarsen for DMD exon 53 skipping, have been approved in the last 4 years. We are witnessing the start of a therapeutic revolution in neuromuscular diseases. However, the studies also made clear that these therapies are still far from a cure. Personalized genetic medicine for neuromuscular diseases faces several key challenges, including the difficulty of obtaining appropriate cell and animal models and limited its applicability. This Special Issue “Molecular Diagnosis and Novel Therapies for Neuromuscular/Musculoskeletal Diseases” highlights key areas of research progress that improve our understanding and the therapeutic outcomes of neuromuscular diseases in the personalized medicine era.

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