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Orphan Disease

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or·phan disease (ôrfn)
n.
A disease that is so rare that it is not considered commercially viable to develop drugs to treat it.

orphan disease
[ôr′fən]
any rare health disorder for which no treatment has been developed. See also orphan drug.

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

orphan disease
Medtalk A disorder affecting < 200,000 people–US–ie, < 1/1000 people. See NICODARD, NORD.


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Because HLH is rare, the federal government has listed it as an orphan disease -- one that affects fewer than 200,000 people -- so it does not qualify for major funding, Toughill said.
ALS is an orphan disease with only about 30,000 patients in the US.
The challenges in the search for SMA therapeutics have led the orphan disease community to new pathways for drug development in neurology.
 
 
 
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