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PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy
Date:4/23/2008

or the treatment of patients with genetic disorders due to a nonsense mutation, a type of mutation found in approximately 13% of patients with DMD. In this double-blind study, patients will be randomized to receive placebo, or one of two dose levels of PTC124, three times per day. Eligible patients will be boys with nonsense-mutation-mediated DMD/BMD who are at least 5 years of age and are able to walk at least 75 meters or approximately 80 yards in six minutes. PTC expects to enroll a total of 165 patients at approximately 35 investigational sites; all study subjects will undergo 48 weeks of blinded treatment. Thereafter, all participants, including those who have been receiving placebo, will be eligible to enroll in an open-label PTC124 extension study.

The primary outcome measure is the total distance walked during a 6-minute walk test, a test of ambulation that has now been standardized for boys with DMD/BMD through a collaboration with noted investigator, Craig McDonald, M.D., at University of California at Davis. Other outcome measures in the Phase 2b study will evaluate activity at home, muscle and heart function, strength, cognitive ability, muscle integrity, and muscle dystrophin expression. Safety parameters, compliance, and PTC124 blood levels also will be monitored.

"We are very pleased to announce the initiation of the Phase 2b trial for PTC124 in boys with DMD/BMD," said Langdon Miller, M.D., Chief Medical Officer of PTC. "We applaud the patients, parents, and clinicians who have committed themselves to this effort. The design of this trial reflects our ongoing collaboration with the advocacy community, investigators at leading neuromuscular centers, and the U.S. and European regulatory agencies. We hope that PTC124 will soon offer a treatment that addresses the underlying cause of the disease for patients with nonsense-mediated DMD/BMD and that the development of PTC124 will set the stage for improving therapeutic options in this disabli
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SOURCE PTC Therapeutics, Inc.
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