Journal article
Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents
Application of clinical genetics, Vol.4, pp.29-44
2011
PMCID: PMC3681176
PMID: 23776365
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.
Details
- Title
- Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents
- Authors/Creators
- Steve D. Wilton - Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, M518, Perth, WA 6009, AustraliaSue Fletcher - Univ Western Australia, Ctr Neuromuscular & Neurol Disorders, M518, Perth, WA 6009, Australia
- Publication Details
- Application of clinical genetics, Vol.4, pp.29-44
- Publisher
- Dove Medical Press Ltd
- Number of pages
- 16
- Grant note
- James and Matthew Foundation Muscular Dystrophy Western Australia; Muscular Dystrophy Association Medical and Health Research Infrastructure Fund, Western Australia National Institutes of Health USA; United States Department of Health & Human Services; National Institutes of Health (NIH) - USA Muscular Dystrophy Association of the United States of America; Muscular Dystrophy Association National Health and Medical Research Council of Australia; National Health and Medical Research Council (NHMRC) of Australia Gavriel Meir Trust
- Identifiers
- 991005592757807891
- Copyright
- © 2011 Wilton and Fletcher
- Murdoch Affiliation
- Centre for Molecular Medicine and Innovative Therapeutics; Health Futures Institute
- Language
- English
- Resource Type
- Journal article
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