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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 ...
An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The ...
The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myoclonus, ALS, Tourette syndrome or muscular dystrophy which affect small numbers of individuals residing in the United States.
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.
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An orphan device is a product or an equipment intended for the prevention, prediction, diagnosis, support, treatment or management of a life-threatening or chronically debilitating disease with a low prevalence/incidence, most notably for rare diseases. [1] Orphan medical technology is then considered as both the medical device (tool or ...
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This was enacted to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myoclonus, amyotrophic lateral sclerosis, Tourette syndrome and muscular dystrophy which affect small numbers of individuals residing in the United States. [29] Not all specialty drugs are orphan drugs.