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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...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Academic Press
2018
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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 |