The new legislation, if passed, would restore the Orphan Disease
Tax Credit that was cut in half by the federal tax bill in 2017.
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.
Rare and Orphan Disease
Center (RODC) at The Jackson Laboratory
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.
[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.
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.
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.)
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
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.
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.
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.