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NIH launches clinical studies nationwide to investigate rare diseases

Contact information for families with rare diseases who hope to participate in the studies are available upon request.The National Institutes of Health (NIH) announced today it is launching the first clinical studies of its Rare Diseases Clinical Research Network (RDCRN). More than 20 studies are expected to open in the next few months at about 50 sites across the United States and in several other countries including the United Kingdom, Japan, and Brazil.

Rare Disease Studies Listed by City: http://www.ncrr.nih.gov/clinical/rdcrn_studylist.asp

Officially, a rare disease is defined as a disease or condition affecting fewer than 200,000 persons in the United States. About 6,000 such disorders have been identified, impacting an estimated 25 million Americans. Few drug companies conduct research into rare diseases since there is little chance to recoup the costs of developing treatments for such small, geographically dispersed populations.

"By studying the genetic component of these rare diseases, we hope to be able to better predict the course of the illnesses and provide more effective, personalized treatments for those afflicted," said Elias A. Zerhouni, M.D., NIH Director. "Ultimately, this individualized approach, completely different from how we treat patients today, will allow us to prevent or to promptly treat the complications arising from these genetic disorders."

The RDCRN has received five-year funding awards totaling $71 million and is coordinated primarily by two NIH components--the Office of Rare Diseases (ORD) and the National Center for Research Resources (NCRR). A central data and technology coordinating center and 10 research consortia will investigate a variety of diseases including Angelman, Rett, Prader-Willi syndromes; myelodysplastic syndrome and other bone marrow failure conditions; lymphangioleiomyomatosis (LAM), rare genetic disorders o
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Contact: Ann Puderbaugh
puderba@mail.nih.gov
301-435-0888
NIH/National Center for Research Resources
5-May-2006


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