MDA, Biotech Company Partner To Work On Cure
Thousands Suffer From Freidrich's Ataxia
POSTED: 1:42 pm EDT September 4, 2009
UPDATED: 1:49 pm EDT September 4, 2009
BOSTON -- MDA has led the world in neuromuscular disease research for nearly 60 years. In that time, scientific technology has grown more costly and more complex.To share in the costs, MDA has formed partnerships with many biotechnology companies. A Massachusetts company is working to find a cure for Freidrich's Ataxia."Repligen is working to develop a novel therapy for treatment of Freidrich's Ataxia. And if we are successful in developing these drugs, it has a potential to be the first drug available and it might modify the course of the disease for the patients and significantly improve the course and trajectory of their life," said Laura Whitehouse, of Repligen.Freidrich's Ataxia is an inherited neurodegenerative disease that is characterized by low levels of the protein Fretaxin, and it leads to progressive disability. It affects the nervous system and muscle function."At Repligen, my responsibility is to develop some methods that we can use to monitor how well our compounds do at increasing the fretaxin gene which is the gene that is under functioning in Freidrich's Ataxia," said scientist Heather Plasterer."We are in the process of developing a drug that we think is actually going to modify FA not just treat the symptoms, but reverse the symptoms. And we are pretty enthusiastic excited about that," Repligen's Steve Jones said.There are about 15,000 patients with Freidrich's Ataxia worldwide and there are currently no therapies available for their treatment."FA is considered an orphan disease and funding from organizations such as MDA are very important for orphan diseases in particular because they are so small and it's very difficult to find the level of funding that you need in order to develop a drug appropriately for patients. One of the key benefits that we have found through the partnership we have with MDA is that it also opens a very extensive network of both physicians and scientists as well as patients all of which are exceedingly valuable in order to help accelerate the development of these compounds for human clinical trials," Whitehouse said.The MDA Labor Day Telethon will be broadcast on Channel 5, starting Sunday at 9 p.m. and continuing on Monday until 6 p.m.
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