Report on Research on Rare Diseases in Children: FY 2000 to FY 2005

摘要

The rare diseases research programs sponsored by NIH Institutes and Centers (ICs) are well-established and well-recognized and include a high percentage of research on children. These basic, clinical, and research training programs continue to contribute to the development and dissemination of information on the prevention, etiology, diagnosis, and treatment of rare diseases in children as well as adults. This report presents the contributions and research advances of the NIH research programs and the Office of Rare Diseases (ORD). Many advances presented in this report are the result of years of basic research sponsored by NIH. Children with rare diseases continue to benefit from the treatment applications realized from the diverse nature of and emphasis placed on both basic and translational research by NIH.