Researchers have begun screening the first definitive collection of thousands of approved drugs for clinical use against rare and neglected diseases. They are hunting for additional uses of the drugs hoping to find off-label therapies, for some of the 6,000 rare diseases that afflict 25 million Americans. The effort is coordinated by the National Institutes of Health’s Chemical Genomics Center (NCGC).

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  1. Therapeutics for Rare and Neglected Diseases Program announces next round of drug development projects Researchers will begin drug development projects for rare and neglected diseases that include potential treatments for a musculoskeletal disorder, a cognitive dysfunction disorder, a virus that affects the central nervous system of newborns, a parasitic worm infection, a form of muscular dystrophy and a rare lung disease....
  2. Rare Disease Day at NIH presents latest research from various agencies and advocacy groups The National Institutes of Health will celebrate the Fourth Annual Rare Disease Day Feb. 28 with a day-long celebration co-sponsored by the NIH Office of Rare Diseases Research and the NIH Clinical Center. The event will recognize rare diseases research activities supported by several government agencies and advocacy organizations. Attendance is free and open to […]...
  3. Researchers identify gene variant in Proteus syndrome A team of researchers has identified the genetic mutation that causes Proteus syndrome, a rare disorder in which tissue and bone grows massively out of proportion. The discovery, which has implications for potential drug therapies and even cancer, appears in the July 27, 2011, early online edition of the New England Journal of Medicine. The […]...
  4. NIH-Supported ACCORD Eye Study Finds Two Therapies Slow Diabetic Eye Disease Progression In high-risk adults with type 2 diabetes, researchers have found that two therapies may slow the progression of diabetic retinopathy, an eye disease that is the leading cause of vision loss in working-age Americans....
  5. Researchers identify genetic cause of new vascular disease Clinical researchers at the National Institutes of Health’s Undiagnosed Diseases Program (UDP) have identified the genetic cause of a rare and debilitating vascular disorder not previously explained in the medical literature. The adult-onset condition is associated with progressive and painful arterial calcification affecting the lower extremities, yet spares patients’ coronary arteries. The new disease finding […]...
  6. What is the difference between prescription drugs and over-the-counter drugs? What is the difference between prescription drugs and over-the-counter drugs...
  7. Medicare, Gene Pattern, May Identify Kidney Transplant Recipients, Who Don’t Need Life-long Anti-rejection Drugs Researchers have identified a distinct pattern of gene expression in the largest reported group of kidney transplant recipients who have not rejected the transplant kidneys even though they stopped taking anti-rejection drugs. This finding may help identify other transplant recipients who could safely reduce or end use of immunosuppressive therapy. In 2008, more than 80,000 […]...
  8. NIH researchers link rare cancer to cell oxygen deficiency Researchers at the National Institutes of Health have discovered that a rare cancer of the digestive tract is linked to a shutdown in an enzyme that helps supply oxygen to cells....
  9. Researchers Identify Gene Mutations Underlying Risk for Most Common Form of Parkinson’s Disease Parkinson's disease, which affects about 1.5 million Americans, is a progressive neurologic disorder caused by the degeneration of nerve cells in the portion of the brain that controls movement....
  10. Medicare, After 40 Years, NIH-Supported Researchers Identify Possible New Treatment for Severe Vasculitis Investigators have made a major advance in treating people with a severe form of vasculitis, anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, a rare but devastating disease of blood vessels. In a six-month study, a new treatment strategy provided the same benefits as the current standard of care used for more than 40 years but required less […]...
  11. Rare gene glitch may hold clues for schizophrenia — NIH-funded study Rare gene glitch may hold clues for schizophrenia -- NIH-funded study...
  12. Researchers identify new marker to predict progressive kidney failure, death A high level of a hormone that regulates phosphate is associated with an increased risk of kidney failure and death among chronic kidney disease (CKD) patients, according to a recent study led by researchers at the University of Miami and funded by the National Institute of Diabetes and Digestive Diseases and Kidney Diseases (NIDDK) at […]...
  13. MedicarCard.com, NIH, Human Microbiome Project, Researchers Publish First Genomic, Collection of Human Microbes The Human Microbiome Project (HMP) today published an analysis of 178 genomes from microbes that live in or on the human body. The researchers discovered novel genes and proteins that serve functions in human health and disease, adding a new level of understanding to what is known about the complexity and diversity of these organisms....
  14. What’s New on the FDA Drugs Site What's New on the FDA Drugs Site...
  15. Medicare: Cholesterol levels elevated in toddlers taking anti-HIV drugs Toddlers receiving anti-HIV drugs have higher cholesterol levels, on average, than do their peers who do not have HIV, according to researchers at the National Institutes of Health and other institutions....