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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Detalhes bibliográficos
Main Authors: Putzeist, Michelle, Mantel-Teeuwisse, Aukje K, Wied, Christine C Gispen-de, Hoes, Arno W, Leufkens, Hubert GM, de Vrueh, Remco LA
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2013
Assuntos:
Research
Acesso em linha: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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