How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration-Reference-Cited by-同舟云学术

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

Published:2015-06-24 Issue:1 Volume:10 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

Furlong Pat,Bridges John F. P.,Charnas Lawrence,Fallon Justin R.,Fischer Ryan,Flanigan Kevin M.,Franson Timothy R.,Gulati Neera,McDonald Craig,Peay Holly,Sweeney H. Lee

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics(clinical),General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13023-015-0281-2.pdf

Reference12 articles.

1. Muscular Dystrophy Association. Duchenne muscular dystrophy: overview [Internet]. Chicago, IL: MDA Inc; http://mda.org/disease/duchenne-muscular-dystrophy/overview Accessed 16 Dec 2014.

2. National Institute of Neurological Disorders and Stroke. Muscular dystrophy: hope through research [Internet]. Bethesda, MD: NINDS; 2014. http://www.ninds.nih.gov/disorders/md/detail_md.htm Accessed 16 Dec 2014.

3. Wicklund MP. The muscular dystrophies. Continuum (Minneap Minn). 2013;19:1535–70. 6 Muscle Disease.

4. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010;9(1):77–93.

5. Centers for Disease Control and Prevention. Prevalence of Duchenne/Becker muscular dystrophy among males aged 5–24 years–four states, 2007. MMWR Morb Mortal Wkly Rep. 2009;40:1119–22.

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