Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents

@inproceedings{Wilton2011NovelCF,
  title={Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents},
  author={Steve D Wilton and Susan Fletcher},
  booktitle={The application of clinical genetics},
  year={2011}
}
The identification of dystrophin and the causative role of mutations in this gene in Duchenne and Becker muscular dystrophies (D/BMD) was expected to lead to timely development of effective therapies. Despite over 20 years of research, corticosteroids remain the only available pharmacological treatment for DMD, although significant benefits and extended life have resulted from advances in the clinical care and management of DMD individuals. Effective treatment of DMD will require dystrophin… CONTINUE READING

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