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Design of a framework for the deployment of collaborative independent rare disease-centric registries: Gaucher disease registry model

Orphan drug clinical trials often are adversely affected by a lack of high quality treatment efficacy data that can be reliably compared across large patient cohorts derived from multiple governmental and country jurisdictions. It is critical that these patient data be captured with limited corporat...

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Detalhes bibliográficos
Publicado no:Blood Cells Mol Dis
Main Authors: Bellgard, Matthew I., Napier, Kathryn R., Bittles, Alan H., Szer, Jeffrey, Fletcher, Sue, Zeps, Nikolajs, Hunter, Adam A., Goldblatt, Jack
Formato: Artigo
Idioma:Inglês
Publicado em: Academic Press 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5729019/
https://ncbi.nlm.nih.gov/pubmed/28190666
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bcmd.2017.01.013
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