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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Bibliographic Details
Published in:Orphanet Journal of Rare Diseases Vol. 13; pp. 1 - 13
Main Authors: Whicher, Danielle, Philbin, Sarah, Aronson, Naomi
Format: research Journal Article
Published: BioMed Central 1/19/2018
Online Access:View this record in EBSCOhost