Rare disease registries: potential applications towards impact on development of new drug treatments

Rare disease registries: potential applications towards impact on development of new drug treatments

Abstract Background Low prevalence, lack of knowledge about the disease course, and phenotype heterogeneity hamper the development of drugs for rare diseases. Rare disease registries (RDRs) can be helpful by playing a role in understanding the course of the disease, and providing information necessa...

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Bibliographic Details
Main Authors: Marijke C. Jansen-van der Weide, Charlotte M. W. Gaasterland, Kit C. B. Roes, Caridad Pontes, Roser Vives, Arantxa Sancho, Stavros Nikolakopoulos, Eric Vermeulen, Johanna H. van der Lee
Format: Article
Language:English
Published: BMC 2018-09-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Rare disease
Registry
Clinical trial
Drug development
Online Access:http://link.springer.com/article/10.1186/s13023-018-0836-0

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http://link.springer.com/article/10.1186/s13023-018-0836-0

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