Drug development for exceptionally rare metabolic diseases: challenging but not impossible

BACKGROUND: We studied to what extent the level of scientific knowledge on exceptionally rare metabolic inherited diseases and their potential orphan medicinal products is associated with sponsors deciding to apply for an orphan designation at the US Food and Drug Administration (FDA) or the Europea...

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Hauptverfasser: Putzeist, Michelle, Mantel-Teeuwisse, Aukje K, Wied, Christine C Gispen-de, Hoes, Arno W, Leufkens, Hubert GM, de Vrueh, Remco LA
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2013
Schlagworte:
Research
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3843583/
https://ncbi.nlm.nih.gov/pubmed/24237580
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-179
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