PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/
Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently. This trial is evaluating the best methods for measuring functional abilities in patients who have lost independent mobility. Patients with nmDBMD develop progressive muscle weakness that leads to deterioration of ambulation, wheelchair dependency, progressive loss of upper limb strength, and eventual respiratory and cardiac failure. The study, which is expected to complete enrollment rapidly, is being funded in part by a
$1 million grant from the Muscular Dystrophy Association, and will involve MDA's five-center DMD Clinical Research Network and a site in the
United Kingdom.
"Given the relentlessly progressive course of DBMD and its impact on multiple organ systems, we believe patients could benefit from therapeutic interventions at all stages of the disease. This trial is an important step in understanding the best methods for evaluating new clinical treatments for these boys and young men," stated Langdon Miller, M.D., Chief Medical Officer at PTC.
"The Muscular Dystrophy Association is proud to provide $1 million in funding towards this ground-breaking study in DBMD patients who have lost the ability to walk," said Valerie Cwik, M.D., Executive Vice President -- Research and Medical Director, Muscular Dystrophy Association. "This trial underscores the importance of MDA's translational research effort. In April 2005, MDA awarded an initial $1.5 million grant to PTC in support of early clinical development of ataluren in nmDBMD. Now we have a trial that will provide valuable information on older DBMD boys and young men that will assist in the design of future clinical trials."