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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...

תיאור מלא

שמור ב:
מידע ביבליוגרפי
הוצא לאור ב: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
פורמט: Artigo
שפה:Inglês
יצא לאור: Academic Press 2018
נושאים:
גישה מקוונת: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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