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New Institute For Rare Diseases

May 22nd, 2009

A unique new institute will look for ways to treat rare and neglected diseases and take the first and riskiest steps toward bringing new drugs to market, U.S. health officials said in a recent report.

According to Reuters news service, the government has provided support for several years to start the Therapeutics for Rare and Neglected Diseases Program at the National Institutes of Health. The program will use taxpayer money to get drugs through the most costly and dangerous phase of development, known as the “Valley of Death” because so many fail there.

The goal is to treat those who suffer from rare conditions that lack treatment. A rare disease is classified as one that affects fewer than 200,000 Americans, and NIH estimates there are about 6,800 of these conditions. Out of this number, only around 200 have treatment.

“Twenty-five to 30 million Americans suffer from rare or neglected diseases,” said acting NIH director Dr. Raynard Kington in a telephone briefing with reporters.

“We don’t know yet exactly which diseases this program will take on,” added Dr. Alan Guttmacher, acting director of the National Human Genome Research Institute.

Guttmacher also said the new institute would be opportunistic, pouncing on promising research studies, some of which may be funded by advocacy groups for rare diseases.

For more information on the Therapeutics for Rare and Neglected Diseases Program, visit:


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