RG2833 is Well-Tolerated and Increases Frataxin Gene Activity

RG2833 is Well-Tolerated and Increases Frataxin Gene Activity. by Amy Madsen on May 17, 2013, Quest Magazine Online (MDA)


Article Highlights:

-Interim results from a clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to act by increasing frataxin gene activity (frataxin protein deficiency is the cause of FA).
-The main aim of the ongoing trial is to assess the safety of RG2833 in people with FA.
-Through its translational research program, MDA has awarded Repligen two grants, totaling more than $1.7 million, to fund development of RG2833 for FA.
-While the encouraging results are an important step forward in finding new drugs, Repligen's Jim Rusche said more tests will be required before a drug like this can be available as a safe and effective treatment for FA.