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A U.S. District Court judge in Newark, N.J., ruled last week that a 16-year-old patient with Duchenne muscular dystrophy should be allowed to use an experimental drug treatment being developed by PTC Therapeutics and currently in clinical trials. The clinical investigator overseeing trials of the South Plainfield, N.J., company's drug PTC124 had determined that the patient, Jacob Gunvalson, did not meet criteria for inclusion in the trial. The suit, brought by the patient's family, contends that the patient was promised treatment. Stuart W. Peltz, CEO of PTC, denies that anyone in the company promised treatment outside of clinical trials. "We believe it is dangerous and counterproductive to allow individual exceptions at this stage of the drug development process," Peltz says. "Delays in completing the FDA-required clinical study or adverse safety information from a protocol exception could slow, or even prevent, approval of PTC124." Pharmaceutical industry consultant James Bruno, president of Chemical & Pharmaceutical Solutions agrees. "It sets a precedent whereby any patient that wants clinical material can sue to get it," he says. Meanwhile, the Phase II data on PTC124 produced in trials on cystic fibrosis treatment conducted at Hadassah Hebrew University Hospital, in Jerusalem, Israel, were published in the Lancet last week. The data indicate a statistically significant improvement in the chloride channel function of patients with nonsense-mutation cystic fibrosis. Cystic fibrosis, muscular dystrophy, and a range of other genetic disorders involve nonsense mutation, which is targeted by PTC124.
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