Research Into Rare Diseases of Childhood

Batshaw, Mark L.; Groft, Stephen C.; Krischer, Jeffrey P.
May 2014
JAMA: Journal of the American Medical Association;5/7/2014, Vol. 311 Issue 17, p1729
Academic Journal
The article focuses on clinical research into rare diseases in children. The role of the Orphan Drug Act of 1983 in the prevalence of clinical trials dealing with rare diseases is discussed. The objectives of the Rare Diseases Clinical Research Network (RDCRN) set up by the U.S. National Institutes of Health (NIH) are enumerated, including the promotion of proof-of concept clinical research projects. Information on the Urea Cycle Disorders Consortium (UCDC) of the RDCRN is presented.


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