Research Into Rare Diseases of Childhood

The acceleration of clinical trials studying rare diseases over the past three decades has occurred largely because of the Orphan Drug Act, which was enacted in 1983 as a result of patient advocacy groups working with congressional sponsors. Rare diseases research in children is of particular import...

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Veröffentlicht in:JAMA : the journal of the American Medical Association 2014-05, Vol.311 (17), p.1729-1730
Hauptverfasser: Batshaw, Mark L, Groft, Stephen C, Krischer, Jeffrey P
Format: Artikel
Sprache:eng
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