Duchenne muscular dystrophy: Drug development and regulatory considerations

Duchenne muscular dystrophy (DMD) is one of the most commonly recognized dystrophinopathies. There are no approved therapeutic options available for this disease but recent discoveries have led to hope that effective therapies might be forthcoming. With funding from patient advocacy groups, private...

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Bibliographic Details
Published in:Muscle & nerve 2010-06, Vol.41 (6), p.740-745
Main Authors: McNeil, D. Elizabeth, Davis, Carole, Jillapalli, Devanand, Targum, Shari, Durmowicz, Anthony, Coté, Timothy R.
Format: Article
Language:English
Subjects:
Androgens - therapeutic use
Animals
Biological and medical sciences
Carbohydrates (enzymatic deficiencies). Glycogenosis
clinical trial design
Clinical Trials as Topic - methods
Clinical Trials as Topic - standards
corticosteroids
Diseases of striated muscles. Neuromuscular diseases
Drug Therapy - standards
Drug Therapy - trends
Duchenne muscular dystrophy
Errors of metabolism
Food and Drug Administration
gene modification therapies
Gentamicins - therapeutic use
Humans
Legislation, Drug - standards
Legislation, Drug - trends
Medical sciences
Metabolic diseases
Muscular Dystrophy, Duchenne - drug therapy
Muscular Dystrophy, Duchenne - genetics
Neurology
Oxandrolone - therapeutic use
Phenotype
Rare Diseases - drug therapy
Research - standards
Steroids - therapeutic use
United States
United States Food and Drug Administration
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Summary:Duchenne muscular dystrophy (DMD) is one of the most commonly recognized dystrophinopathies. There are no approved therapeutic options available for this disease but recent discoveries have led to hope that effective therapies might be forthcoming. With funding from patient advocacy groups, private investors, and governmental bodies such as the Food and Drug Administration Office of Orphan Product Development (FDA/OOPD), gene modification and other molecular therapies are being actively investigated. However, since DMD patients are few in number and disease manifestations vary considerably in early and late stages of disease, obtaining the data needed for full evaluation of putative therapies may prove challenging. Should ambulation remain the focus of Phase 2/3 studies or should consideration be given to the primary causes of late‐stage morbidity and mortality, e.g., cardiac and respiratory dysfunction related to reduced or absent dystrophin production? It seems reasonable to argue that clinical trials planned for DMD should consider the entire population. Muscle Nerve, 2010
ISSN:0148-639X
1097-4598
DOI:10.1002/mus.21623