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Rare Diseases & Conditions

A rare disease is a condition that affects fewer than 200,000 people, as defined by the U.S. Congress in the Orphan Drug Act of 1983. Although each rare disease affects a small number of individuals, the total number of individuals suffering from rare diseases is in fact high. There are approximately 7,000 rare diseases, affecting a total of 25 to 30 million people in the United States.

Many rare diseases are chronic and debilitating, with effective therapies not yet available to patients. Researchers at the Li Weibo Institute for Rare Diseases Research collectively work on approximately 60 different rare diseases, some of which are listed below. This research is revealing molecular mechanisms, identifying novel causative genes, and developing potential curative therapies for specific rare diseases, as well as improving gene therapy tools for application to a broad range of rare diseases. Select a rare disease from the list for more information about current research, therapeutic strategies, and clinical trials.