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

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 necessary for c...

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Detalles Bibliográficos
Autores principales: Jansen-van der Weide, Marijke C., Gaasterland, Charlotte M. W., Roes, Kit C. B., Pontes, Caridad, Vives, Roser, Sancho, Arantxa, Nikolakopoulos, Stavros, Vermeulen, Eric, van der Lee, Johanna H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6126025/
https://www.ncbi.nlm.nih.gov/pubmed/30185208
http://dx.doi.org/10.1186/s13023-018-0836-0

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