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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Autors principals: Putzeist, Michelle, Mantel-Teeuwisse, Aukje K, Wied, Christine C Gispen-de, Hoes, Arno W, Leufkens, Hubert GM, de Vrueh, Remco LA
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2013
Matèries:
Research
Accés en línia: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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