orphan disease

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or·phan dis·ease

a disease for which no treatment has been developed because of its rarity (affecting no more than 200,000 persons in the U.S.).
See also: orphan products.

Farlex Partner Medical Dictionary © Farlex 2012

orphan disease

A disease that is relatively rare, for which the development of drugs is considered to be commercially nonviable.

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.

or·phan dis·ease

(ōr'făn di-zēz')

A disease for which no treatment has been developed because of the disorder's rarity.
See also: orphan products

Medical Dictionary for the Health Professions and Nursing © Farlex 2012

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The new legislation, if passed, would restore the Orphan Disease Tax Credit that was cut in half by the federal tax bill in 2017.

Gottheimer invites Closter resident as State of the Union guest

Ideally, you'd like to sample many unrelated patient samples with the same mystery condition or orphan disease, and find the same (or very similar) non-host sequence appearing in a large proportion of the samples.

Pathogen discovery through deep sequencing

Rare and Orphan Disease Center (RODC) at The Jackson Laboratory

Orphan drugs: rare diseases, rare funding: institutions dedicated to finding treatments for rare diseases employ unique R&D methods

However, there are number of issues and barriers unique to developing treatments for orphan diseases that pharmaceutical companies face and which are often not well understood by the general public.

We need new approach to orphan medicines

[USPRwire, Sun Jul 26 2015] The Global Orphan Diseases Partnering 2010-2015 report provides an understanding and access to the orphan diseases partnering deals and agreements entered into by the worlds leading healthcare companies.

Global Orphan Diseases Partnering 2010-2015: Deal trends, players and financials - New Market Report

18 June 2013 - Several pharmaceutical firms have shown interest in buying US biotechnology company ViroPharma Inc (NASDAQ:VPHM), although the rare and orphan disease treatment maker is not looking for a buyer, three insiders told Reuters.

Several drug makers reportedly eye ViroPharma

Xechem believes that the inhibiting effect of leupeptin and the targeting impact of carnitine make Myodur an ideal candidate for this "orphan disease." (Ceptor previously received orphan drug status for the use of leupeptin as a therapeutic treatment for nerve injuries.)

Orphan Drug incentives produce results

He believes the alliance is a "clear positive for Alnylam "dramatically expanding" its RNAi drug discovery capabilities and strengthening its balance sheet to advance its own orphan disease programs.

Alnylam alliance with Regeneron a 'clear positive,' says Piper Jaffray

Despite the challenges of enrolling patients into an orphan disease study, Pharm-Olam completed enrollment for the HERCULES study ahead of schedule, the team managed 92 sites across 16 countries to dose 145 patients.

Pharm-Olam Conducts Pivotal Orphan Disease Trial, Leads to First Therapeutic Approved for the Treatment for Acquired Thrombotic Thrombocytopenic Purpura

Massachusetts-based Lotus Tissue Repair is the developer of a protein replacement therapy that is currently being investigated for the treatment of dystrophic epidermolysis bullosa (DEB), an orphan disease for which there is no approved treatment option yet.

Ireland's Shire to acquire US biotech firm Lotus Tissue Repair

Inovio plans to further develop INO-3106 as a novel non-invasive immunotherapy for the treatment of RRP, a rare, orphan disease, for both adult and pediatric populations.This data was presented at this week's Annual Meeting of American Association for Cancer Research.