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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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Detalles Bibliográficos
Autores principales:	Putzeist, Michelle, Mantel-Teeuwisse, Aukje K, Wied, Christine C Gispen-de, Hoes, Arno W, Leufkens, Hubert GM, de Vrueh, Remco LA
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2013
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3843583/ https://www.ncbi.nlm.nih.gov/pubmed/24237580 http://dx.doi.org/10.1186/1750-1172-8-179

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