[The basic concept of therapeutic approaches for DMD].

  • Helge Amthor
  • Published 2015 in
    Archives de pediatrie : organe officiel de la…


Duchenne muscular dystrophy (DMD) is the most frequent hereditary neuromuscular disorder in childhood. Over the past 30 years, increasingly better standards of care have considerably improved the quality of life as well as the life expectancy of DMD patients. Despite such progress in disease management, DMD remains a devastating disorder with continuous decline of motor and cardiac function. Until recently, corticosteroids were the only treatment available to slow down, however modestly, disease progression. Importantly, novel innovative therapeutic approaches are currently being developed. This review discusses the rational and underlying molecular mechanism of these novel strategies as well as the progress made by recent clinical trials. Importantly, these new therapeutic advances bear the potential to profoundly modify the disease course of DMD.

DOI: 10.1016/S0929-693X(16)30011-2

Cite this paper

@article{Amthor2015TheBC, title={[The basic concept of therapeutic approaches for DMD].}, author={Helge Amthor}, journal={Archives de pediatrie : organe officiel de la Societe francaise de pediatrie}, year={2015}, volume={22 12 Suppl 1}, pages={12S63-8} }