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

Authors Steve D Wilton, Sue Fletcher

Published 10 March 2011 Volume 2011:4 Pages 29—44

DOI https://doi.org/10.2147/TACG.S8762

Review by Single-blind

Peer reviewer comments 4

Steve D Wilton, Sue Fletcher
Centre for Neuromuscular and Neurological Disorders, University of Western Australia, Crawley, Perth, WA, Australia

Abstract: 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 restitution in skeletal, cardiac, and smooth muscles and nonmuscle tissues; however, modulation of muscle loss and regeneration has the potential to play an important role in altering the natural history of DMD, particularly in combination with other treatments. Emerging biological, molecular, and small molecule therapeutics are showing promise in ameliorating this devastating disease, and it is anticipated that regulatory environments will need to display some flexibility in order to accommodate the new treatment paradigms.

Keywords: Duchenne muscular dystrophy, molecular therapeutics, small molecules

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