Orphan diseases Drugs

Epilepsy affects approximately 1 percent of the human population, with an estimated 50 million people worldwide currently suffering from the disorder. Mayo Clinic and partners from the University of Pennsylvania School of Veterinary Medicine, the University of Minnesota College of Veterinary Medicine and College of Pharmacy, the Perelman School of Medicine at the University of Pennsylvania, and NeuroVista Corporation have been awarded $7.5 million grant (U01) from the National Institute of Neurological Disorders and Stroke, a division of the National Institutes of Health (NIH). The research involves studying new ways to predict and control epileptic seizures in dogs and people.

While some bear names you’ve likely never heard of, other examples may surprise you. Huntington’s disease, Tourette’s syndrome and muscular dystrophy are all classified as rare diseases. In all, more than 7, 000 medical disorders are considered rare.

For decades, Americans diagnosed with rare diseases had little hope. Drugs used to treat such conditions – known as “orphan drugs” – often never made it out of the lab as prioritization was put on drugs that would help conditions affecting millions. Then, in 1983, Congress passed the Orphan Drug Act in order to stimulate the development of new treatments for rare diseases. The move worked; since 1983 more than 350 new drugs have been developed.

Today, the University of Minnesota Center for Orphan Drug Research is investing new resources into the development of orphan drugs and drug delivery systems that could impact the lives of millions of people both in Minnesota and across the United States.

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Popular Q&A

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What is medical definition of orphan disease?

Any disorder affecting less than 200,000 people in the US (less than one per 1,000 people)—regarded by the pharmaceutical industry as too rare for developing commercially viable products

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