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An overview of the impact of rare disease characteristics on research methodology

BACKGROUND: About 30 million individuals in the United States are living with a rare disease, which by definition have a prevalence of 200,000 or fewer cases in the United States ([National Organization for Rare Disorders], [About NORD], [2016]). Disease heterogeneity and geographic dispersion add t...

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Detalles Bibliográficos
Autores principales: Whicher, Danielle, Philbin, Sarah, Aronson, Naomi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5775563/
https://www.ncbi.nlm.nih.gov/pubmed/29351763
http://dx.doi.org/10.1186/s13023-017-0755-5