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Sarepta Therapeutics, Inc. Message Board

  • bendestre bendestre Apr 8, 2013 6:08 PM Flag

    There is no double blind randomized placebo controlled trial showing eteplirsen improves walk

    In God we trust.
    Everyone else brings data.
    Still waiting.
    If anyone on this board has information to the contrary please come forward.
    No insults and ranting please. Just hard facts and data.

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    • If you think a placebo effect can get DMD kids to perform in the 6 minute walking test without declines in test performance over a year and a half, then you just don't understand DMD. Moreover, with the crossover design of the eteplirsen trial, there is a second trial arm with clear evidence of clinical benefit from the 4 kids whose eteplirsen treatment was delayed.

      The drug and the exon skipping technology work.

    • You are most likely correct so you should go short - HUGE!

    • You are the one that is blind to the facts - Fact #1 - no DMD patient ever has or ever will begin sponaneously producing dystrophin, so the placebo aspect of dystrophin production measures is meaningless. Fact #2 - the lack of dystrophin is clinically accepted as the reason patients lose muscle strength, so dystrophin production as a surrogate endpoint is a lock. Fact#3 - any holes you want to shoot in the 6MWT results are meaningless, because they don't have to prove clinical effectiveness in order to get accelerated approval. All your ranting about controlled trials don't mean a thing in the rare disease space - the FDA has enormous flexibility in determining the evidence that can be considered in an accelerated approval filing. While you focus on two patients that deteriorated prior to their dystrophin levels being impacted, there are 10 others that are stabilizing on their 6MWT measures and as the FDA official noted in a recent presentation, "patient reported outcomes" (i.e. anecdotal evidence on how the drug has impacted the patients' lives) is a very important consideration in accelerated approval filings with limited patient populations. Combine that with a pristine safety profile and accelerated approval is a slam dunk. Time for you to move on.

    • With what we know, a double blind, placebo controlled data will never forget about it. A parent would never sign off on a death sentence, especially in light of our safety data, dystrophin production and 6MWT. This trial design will NEVER happen going forward. So quit wasting your time. For a life time, these kids have already received hope or therapy...we already know the results.

    • If you need double blind data for a drug that treats DMD then you shouldn't be investing in biotech.

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