What clinical trials have you already conducted in Duchenne/Becker muscular dystrophy and what were the results?
After successfully completing two Phase 1 clinical trials in healthy volunteers, we started a Phase 2a trial in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) at three sites in the U.S. Each of the 38 patients in this trial received ataluren for 28 days at one of three dose levels. The goal of this study was to determine whether ataluren increases the production of dystrophin in muscle cells. DMD is caused by a lack of dystrophin protein. Because ataluren is designed to increase the production of dystrophin in patients with nmDMD, we monitored dystrophin levels in muscle cells. This was accomplished by removing a small amount of muscle tissue from each participant in the trial and analyzing it using a microscope and specialized software. A lack of dystrophin in muscle cells causes the protein creatine kinase to leak out of muscle cells into the bloodstream. Thus, another goal of this study was to determine whether ataluren reduces the concentration of creatine kina
