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Sarepta Therapeutics, Inc. Message Board

  • bcdmyers43 bcdmyers43 Oct 9, 2013 7:02 PM Flag

    Skipping doesn't work. Doesn't really work very well in theory

    And sure the heck doesn't work in a human. Better plays are immunotherapy and antisense like Isis. Gene silencing via double stranded RNA degradation.

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    • Doesn't matter how it works in theory because it works damn good in reality.

    • It's not only the Etep. boys who would be faking then, according to you, but also some of the boys who were on the GSK Drisapersen trial. Actually there are reports from the families of these boys that the drug appeared to be working on them. A collective placebo effect? unlikely, as there are also indication from the some specific statistics that the drug had an effect. The problem eas the risk/benefit ratio, as GSK stated.

      Actually, the reason GSK has put the drug on hold seems to be mainly related to its safety profile: they had problems dosing the drug at levels such as to reach target therapeutics effects on the sample of patients as a whole. In other words, there were dose-limiting issues, due to the safety of the drug. I am just repeating here what Redplate said from the onset (long time ago). Since Etep. does not have these safety issues, it can be dosed at levels possibly likely to reach desired therapeutics effects.

      Therefore, the issue should not be if exon skipping works or not (Kole said in Naples theat Etep. has high target specificity), but rather the degree to which the drug is able to reach its target (the intake of the drug). Future will tell . So far, so good though, based on distrophyn evidence and from 6MWD results. IMHO.

    • so the kids who have maintained their ambulation are faking or are experiencing placebo effects - is that what you're saying? I don't think dmd sufferers can fake ambulation

    • You are a SHORT joke!

      Sentiment: Buy

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