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Biennial and Annual Report on the Rare Diseases Research Activities at the National Institutes of Health FY 2004

NIH Report on Research on Rare Diseases in Children, FY 2000 to FY 2005

2001 Report on the Special Emphasis Panel of the National Institutes of Health on the Coordination of Rare Diseases Research

Report on Scientific Workshops and Symposia as a Mechanism to Stimulate Research on Rare Diseases and Conditions, 1997

Report of the National Commission on Orphan Diseases - February 1989
Appendices - Volume I
Appendices - Volume II

1979 Interagency Task Force Report on Significant Drugs of Limited Clinical Value

IOM Report-Rare Diseases and Orphan Products: Accelerating Research and Development
Rare diseases collectively affect millions of Americans of all ages. They often are serious and life altering; many are life threatening or fatal. Because each rare disease affects a relatively small population, however, it can be challenging to develop drugs and medical devices to prevent, diagnose, and treat these conditions. We still lack even a basic understanding of the cause or underlying molecular mechanisms of many rare diseases. To help in accelerating rare diseases research and product development, the National Institutes of Health (NIH), with support from the Food and Drug Administration (FDA), asked the Institute of Medicine (IOM) to examine the opportunities for and obstacles in developing drugs and medical devices for treating rare diseases.


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