Jim Rusche from Repligen Corp. announced encouraging interim results from a phase 1 clinical trial of RG2833 in Friedreich's ataxia
posted on May 17, 2013 - 4:39pm
Interim results from a phase 1 clinical trial of RG2833 in people with Friedreich’s ataxia (FA) show that the experimental drug is well-tolerated, and that it appears to increase the activity of the gene for the frataxin protein.
A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake
posted on September 30, 2009 - 1:28pm
Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experimental compound being developed to treat this disease. MDA is supporting Repligen to develop this...