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A rare disease is any disease that affects a small percentage of the population. In some parts of the world, the term orphan disease describes a rare disease whose rarity results in little or no funding or research for treatments, without financial incentives from governments or other agencies. Orphan drugs are medications targeting orphan ...
The bipartisan Rare Disease Legislative Caucus was established in 2009 by original GOP Co-chair Congressman Fred Upton (R-MI), who went on to become Chairman of the House Energy and Commerce Committee, and Congressman Joseph Crowley (D-NY). Jurisdictional issues, including Food and Drug Administration oversight, as well as a myriad of issues ...
Fields condition, [1] also known as Fields' disease, [2] [3] is a neuromuscular disease that is considered the rarest medical condition in the world. It was named after Welsh identical twins Catherine and Kirstie Fields, who are two of only three people known to have been affected.
Any disease that impacts 200,000 people or less in the U.S. is classified as a rare disease. The rare disease community is a close-knit group of patients, parents and specialists determined to ...
The following is a list of genetic disorders and if known, type of mutation and for the chromosome involved. Although the parlance "disease-causing gene" is common, it is the occurrence of an abnormality in the parents that causes the impairment to develop within the child.
Orphanet is an online database with the goal of gathering, providing and improving knowledge on rare diseases and to improve the diagnosis, care and treatment of patients with rare diseases. By listing rare diseases, and maintaining a standard nomenclature of rare diseases (ORPHAcodes), Orphanet contributes to making them more visible in health ...
There are more than 6,000 known rare diseases, and it is estimated that about 25 million Americans are affected by them (as of 2002). [2] Prior to the RDA was the Orphan Drug Act of 1983, which was designed to facilitate the development and commercialization of drugs to treat rare diseases, termed orphan drugs. This act, however, did not ...
An ultra-rare disease is a disease that affects an extremely small percentage of the population. In some parts of the world, an ultra-orphan disease is a rare disease whose rarity means there is a lack of a market large enough to have support and resources for discovering treatments for it. [1] [2] [3]