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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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Detalles Bibliográficos
Autores principales: Bellgard, Matthew I., Napier, Kathryn R., Bittles, Alan H., Szer, Jeffrey, Fletcher, Sue, Zeps, Nikolajs, Hunter, Adam A., Goldblatt, Jack
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Academic Press 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5729019/
https://www.ncbi.nlm.nih.gov/pubmed/28190666
http://dx.doi.org/10.1016/j.bcmd.2017.01.013