New article on rare diseases

[annekat]

Rare diseases - pharma?s new frontier | InPharm (http://www.inpharm.com/news/171918/rare-diseases-pharma-s-new-frontier)

This is a tidbit from the Vasculitis Education "group" on Yahoo. Many of the articles are pretty technical but this one is pretty readable for non-eggheads like me. It is more from the point of view of the "Pharma" companies and what they are doing about treatments for rare diseases in the US and the UK. WG and MPA are mentioned, as is Rare Disease Day. I thought it might interest some of you.

[mr.g]

Thanks for the article. It is good news that the rare disease science community is again trying to organize their efforts around the world. At least it sounds good. Time and money or is it money and time will help? I will remember Feb.29 next year. Thanks again....

[drz]

Quote from above article on WG and MPA--note not GPA in article yet?

Roche is using its biologics expertise, especially through its subsidiary Genentech, to make in-roads in the rare diseases field. In April 2011 Roche and Biogen Idec gained US approval for the use of Rituxan/MabThera (rituximab), in combination with corticosteroids, as a new medicine for adults with Wegener’s Granulomatosis (WG) and Microscopic Polyangiitis (MPA).

WG and MPA are two severe forms of vasculitis called ANCA-Associated Vasculitis (AAV), a rare autoimmune disease that largely affects the small blood vessels of the kidneys, lungs, sinuses, and a variety of other organs. Both WG and MPA are considered orphan diseases with an estimated prevalence in the US of approximately three cases per 100,000 people.

[annekat]

Yeah, I wonder, too, why no mention of GPA, since the article just came out. Also I'm not sure what they mean by an "orphan disease".... yeah, they are rare diseases, but how they got that name.... I guess orphans are pretty rare.