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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 ...
A rare disease is technically defined (in the European Union) as a disease that is found in fewer than 5 people per every 10,000 people. ...
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 ...
There are few times when your doctor or physician will utter the phrase, "I don't know" to a patient, but when you're dealing with the rarest of diseases, then all bets are off. According to the ...
The National Organization for Rare Disorders (NORD) was established in 1983 by individuals and families with rare diseases. [42] [43]EveryLife Foundation for Rare Diseases was founded in 2009 and is a nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable ...
Orphanet is an organisation and knowledge base dedicated to rare diseases as well as corresponding diagnosis, orphan drugs, clinical trials and expert networks. [ 1 ] Orphanet was founded in France in 1997 by Inserm , the French National Institute of Health and Medical Research.
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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] Distinct countries define and provide special economic incentives for companies developing drugs that treat ultra-rare diseases.
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