Muscular dystrophy: new challenges and review of the current clinical trials

Curr Opin Pediatr. 2013 Dec;25(6):701-7. doi: 10.1097/MOP.0b013e328365ace5.

Abstract

Purpose of review: We provide a review of recent standards of care and therapeutic development in different forms of muscular dystrophies. This topic is relevant as the improved understanding of these disorders has not only led to a better definition of clinical course and to the development of standards of care for individual types of muscular dystrophies, but also culminated in different therapeutic approaches.

Recent findings: Recent natural history studies have demonstrated the impact of new standards of care in different forms of muscular dystrophies, and identified areas of clinical management in which further developments are needed. The majority of the experimental studies are focused on Duchenne muscular dystrophy. Some of them target patients with specific mutations, such as antisense oligonucleotides, to induce exon skipping of specific mutations or drugs developed to allow read-through of nonsense mutations, whereas other therapies deal with secondary aspects of muscle degeneration, aiming, for example, at reducing inflammation or apoptosis, and may also be suitable for other forms of muscular dystrophies.

Summary: The advances in the field of muscular dystrophy have resulted in improved clinical course and survival. The encouraging results of early experimental studies could further improve these outcomes in the future.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adolescent
  • Angiotensin-Converting Enzyme Inhibitors / therapeutic use*
  • Anti-Inflammatory Agents / therapeutic use*
  • Child
  • Child, Preschool
  • Clinical Trials as Topic
  • Codon, Nonsense / drug effects*
  • Codon, Nonsense / genetics
  • Female
  • Genetic Predisposition to Disease
  • Genetic Therapy
  • Humans
  • Infant
  • Male
  • Muscular Dystrophies / drug therapy*
  • Muscular Dystrophies / genetics*
  • Oligoribonucleotides, Antisense / therapeutic use
  • Translational Medical Research

Substances

  • Angiotensin-Converting Enzyme Inhibitors
  • Anti-Inflammatory Agents
  • Codon, Nonsense
  • Oligoribonucleotides, Antisense