Interventions for muscular dystrophy: molecular medicines entering the clinic

  title={Interventions for muscular dystrophy: molecular medicines entering the clinic},
  author={K. Bushby and Hanns Lochm{\"u}ller and S. Lynn and V. Straub},
  journal={The Lancet},
  • K. Bushby, Hanns Lochmüller, +1 author V. Straub
  • Published 2009
  • Medicine
  • The Lancet
  • Muscular dystrophies are individually rare genetic disorders that cause much chronic disability, affecting young children and adults. In the past 20 years, more than 30 genetic types of muscular dystrophy have been defined. During this time, precise diagnosis, genetic counselling, and medical management have improved. These advances in medical practice have occurred while definitive therapies based on an improved knowledge of disease pathogenesis are awaited. A wide range of therapeutic options… CONTINUE READING
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