Concepedia

Clinical research for rare disease: Opportunities, challenges, and solutions

Robert C. Griggs, Mark L. Batshaw, Mary Dunkle, Rashmi Gopal-Srivastava, Edward M. Kaye, Jeffrey P. Krischer, Tan T. Nguyen, Kathleen Paulus, Peter A. Merkel

Molecular Genetics and Metabolism · 2008 · 407 citations · 13 references

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13

The Design and Analysis of Clinical Experiments.

Michael Hills, Joseph L. Fleiss · Journal of the Royal Statistical Society Series A (General) · 1987 · 3.3K citations

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A recurrent mutation in the BMP type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva

Eileen M. Shore, Meiqi Xu, George Feldman et al. · Nature Genetics · 2006 · 1.2K citations

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Randomized versus historical controls for clinical trials

Henry S. Sacks, Thomas C. Chalmers, Harry Smith · The American Journal of Medicine · 1982 · 837 citations

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Determining Optimal Therapy — Randomized Trials in Individual Patients

David L. Sackett, D. Wayne Taylor, John Ghong et al. · New England Journal of Medicine · 1986 · 587 citations

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Randomized phase II clinical trials.

Richard Simon, R. Wittes, Susan S. Ellenberg · PubMed · 1985 · 470 citations

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