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Sarepta Therapeutics, Inc. Message Board

  • musherga musherga Feb 12, 2013 5:31 PM Flag

    placebo effect/unblinding

    This point has been made here but the media seems to miss it. The primary endpoint is a marker for dystrophin. There's no placebo effect for biochemical response to treatment. The 6 minute walk test is a secondary end point. Each boy in the test serves as his own control. A decine in the rate of decay at the stage of disease chosen for the study would be a positive outcome. This is a progressively debilitating disease. A placebo effect, i.e. psycho-biological improvement, cannot be sustained in the face of progressive muscle wasting. This isn't a headache pill.

    Also the truncated dystrophin produced by restoring the reading frame produces a protein that carries a highly variable degree of muscle impairment. It is expected that some patients will not benefit greatly, perhaps slowing progression, or won't benefit at all. Others are expected to see dramatic improvement. So far no evidence has shown that the drug made boys worse. Early approval is for the boys that might go from riding a wheelchair to playing soccer by taking it. These same boys can look forward to spinal fusion, and ventilator dependency without it.

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