Are any ataluren clinical trials open for patient enrollment?
Yes. We are conducting Phase 2a clinical trials to evaluate the safety and activity of ataluren in patients with nonsense mutation hemophilia type A and B (nmHA/nmHB) and with nonsense mutation methylmalonic acidemia (nmMMA). The Phase 2a trial of ataluren in patients with nonsense mutation nmHA/nmHB is being conducted at multiple sites in the U.S., Canada, France and Italy. The main goals of the trial are to determine the effect of ataluren administration on Factor VIII or IX levels and whether or not the drug can be given safely to people with severe hemophilia due to a nonsense mutation. The trial is open to males at least 18 years of age with nmHA/nmHB who are not receiving or are willing to forgo prophylactic factor during the trial, which lasts about six weeks. For more information about the trial and a list of sites, please see the hemophilia clinical trial overview. [link]. People with nmHA/nmHB or their families should contact a site directly to inquire about trial participati
