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Clinical research for rare disease: opportunities, challenges, and solutions.
Clinical research for rare disease: opportunities, challenges, and solutions.
R. Griggs,M. Batshaw,6 Auteurs,P. Merkel
2009 · DOI: 10.1016/j.ymgme.2008.10.003
Molecular Genetics and Metabolism · 358 citations
TLDR
The scientific methodology, financial resources, and logistics of clinical research for rare diseases have changed dramatically in the past two decades resulting in increased understanding of the pathophysiology of these disorders and direct benefit to patients.