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Sarepta Therapeutics, Inc. Message Board

  • simp08801 simp08801 Oct 5, 2013 7:17 AM Flag

    GSK: words to RNA trial parents that matter in CAPS

    "Overall, the safety profile of Study DMD114044 is consistent with that seen in the other drisapersen studies. Based on the preclinical and clinical safety profile of drisapersen, adverse events were predictable, YET NOT TRIVIAL. As expected,subclinical proteinuria was one of the most commonly reported adverse events after injection site reactions. Subclinical proteinuria was reported in 57/125 (46%) boys treated with drisapersen and 15/61 (25%) boys treated with placebo. As previously reported, one boy had severe proteinuria requiring hospitalization to facilitate treatment and recovery. This boy fully recovered following withdrawal of drisapersen and appropriate treatment. No boys had thrombocytopenia during the phase III DMD114044
    study, however in tracking participants from other studies with LONGER TERM EXPOSURE to the medicine SOME cases of SEVERE thrombocytopenia were seen."

    so now you know

    the longer term extension of the GSK P2 (i.e., longer than the P3) has resulted in "some" cases of "severe" thrombocytopenia

    I assure you that when a pharma says "some" in this context, its much more than, say, 2

    worse yet, when a pharma uses "severe" in this context, it means horrific

    drisaperson is a drug that is, by definition, a LIFETIME drug

    these disclosures to the tRNA trial parents give you a glimpse of a tip of an iceberg

    I cannot imagine GSK will continue with this

    "some" and "severe" are the words that make me believe this

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