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Vertex Pharmaceuticals Incorporated Message Board

algeaqueen 5 posts  |  Last Activity: May 7, 2016 1:52 PM Member since: Dec 2, 2011
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  • Some highlights of the large study:
    When given the choice between managing symptoms or minimizing side effects, nearly three-quarters of surveyed adults with schizophrenia who have ever taken schizophrenia medication (70 percent) and surveyed caregivers whose loved one has ever taken schizophrenia medication (74 percent) report that it is more important to ‘manage symptoms.’ However, more than half of these adults with schizophrenia (51 percent) and caregivers (56 percent) report feeling frustrated with schizophrenia medication because the side effects are hard to deal with. Survey results imply, however, that the trade off these adults with schizophrenia and caregivers may be making does not result in complete symptom management: nearly three in four (73 percent) surveyed adults with schizophrenia who currently take medication report still dealing with schizophrenia symptoms at least monthly, and almost nine in ten (87 percent) surveyed caregivers whose loved one currently takes medication report the same.

    As they work to help patients manage the symptoms of the disease, psychiatrists surveyed report feeling frustrated by medication side effects (90 percent), compliance (80 percent) and efficacy (66 percent)

    Only 20 percent of psychiatrists surveyed agree that most of their schizophrenia patients always take their medication exactly as prescribed, and 90 percent worry that their patients will not take their medication exactly as prescribed. These psychiatrists cite side effects as the number one thing that frustrates their patients about their schizophrenia medication.

    *Disclosure: Long ITCI @31. I anticipate a bidding war for this company on successful completion of their Phase 3 trial in schizophrenia as ITI-007 could be the ONLY drug for this disease as well as Bi-polar disease(an even larger patient population) which does not cause side effects. That is worth at least several billion to an AGN or LLY imo.

    Sentiment: Strong Buy

  • Society presentation in October 2013, about 20 percent of boys older than 7 lost their ability to walk after just one year. At that rate, more than half (about 59 percent) would have been unable to walk after four years.
    3. The FDA’s argument that boys in Sarepta’s trial received more steroids than the boys used for its control group appears wrong. Jenn McNary, a mother of a boy in the trial, already made this argument last Friday, saying her son was underdosed with steroids during the course of the trial. But there’s evidence in Sarepta’s application itself to support this (specifically, in Appendix 3). The actual baseline doses of steroids are listed (some are in milligrams, while others are in milligrams per kilogram). Converting them all to milligrams per kilogram, and averaging each arm, the boys in Sarepta’s trial received an average baseline dose of 0.79, less than the average of boys in the control group of 0.81.
    4. Likewise, the agency’s argument that the boys in the control group were given steroids intermittently (rather than continuously) is not as strong as it appears. On page 26 of the briefing documents the agency says that 69 percent of the boys in the control group got steroids intermittently. But the same chart I referred to above lists five out of 13, or 38 percent, of the boys on “intermittent” steroid dosage. And there is evidence to argue that even that is not a big deal: A 2011 paper in the journal, Neurology, found that weekly dosing of Duchenne patients with steroids was just as effective as daily dosing.

  • I've read that the shorts believe the SRPT patients have mostly benefited from steroid treatment rather than Etep. Were the control patients, who showed loss of ambulation, also treated with steroids?

  • algeaqueen algeaqueen Apr 4, 2016 3:05 PM Flag

    WWAV will likely become an acquisition target by the end of the year imo

    Sentiment: Strong Buy

  • which is basically meaningless, He's still positive on the shares and didn't reduce his price target.

    Sentiment: Strong Buy

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