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Sarepta Therapeutics, Inc. Message Board

  • simp08801 simp08801 Apr 1, 2013 8:36 AM Flag

    the anecdotal evidence continues to leap

    at this point the bull and bear competing articles are almost meaningless

    seems to me that the best thing to do, by a long shot, is to simply follow the anecdotal evidence

    the importance of the following quote from last week CANNOT be overstated:

    "During our meeting, she relayed anecdotal stories about the remarkable
    benefits of eteplirsen in many of the 12 subjects in the eteplirsen study including many
    that cannot be captured by the six-minute walk test in young children, including balance,
    fine motor skills, higher energy, new motor skills, as well as cardiac and cognitive

    I repeat, "MANY"

    you may not yet realize it, but you already KNOW what the 74 week data will show when released

    and remember, the original placebo group was on drug for 48 weeks, as of week 72 of the trial

    and I am sure that is why the word "MANY" was used

    there is a stockpile of public evidence available to weigh the risk/reward

    and it grew materially just last week

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    • BTW, Is there a web conf for that Conference in DC?

    • The 72 week must be good because anything else would have been a material event that would require a press release.I think also the backing off of the dmd parents after their meeting also looks to be a factor.

    • and I would keep an eye on the World Orphan Drug Conference on April 9-11 in DC

      sarepta is a sponsor

      if you remember the CEO's public comments about the appropriate scientific venue to release the 74 week data, this may be it

      I believe this is going to be a groundbreaking month

      more anecdotally, public comments by sarepta trial parents that there were TWO 6m walk tests is also anecdotally telling

      the 84 week sarepta tests entailed ONE test

      the other test related to the Florida based mri study, where additional data is obviously continuing to be collected

      when will that data be released and what will it show?

      I would not want to be on the "wrong" side of that data either

      • 4 Replies to simp08801
      • Just a heads up, but there are always two 6MWT tests at each interval. The company then takes the better score of the two tests. They did this at baseline and with placebo group as well, so as not to skew results.

      • Chris Garabedian, CEO of Sarepta Therapeutics will be presenting "Leveraging a Personalized Medicine Platform to Treat Genetic Sub-Groups " at The World Orphan Drug Congress USA 2013 in Washington D.C. (April 9-11, 2013). In this exclusive conversation with Rare Disease Report, Mr. Garabedian previews his presentation.

      • RNA and Oligonucleotide Therapeutics
        April 10 - 13, 2013
        Abstract Deadline: March 1, 2013

        Ryszard Kole, Sarepta Therapeutics
        Adrian Krainer, Cold Spring Harbor Laboratory
        Art Krieg, RaNA Therapeutics
        Bruce Sullenger, Duke University

        We are pleased to announce the third Cold Spring Harbor conference on RNA and Oligonucleotide Therapeutics, which will begin at 7:30 pm on Wednesday April 10 and run through lunch on Saturday, April 13, 2013.

        Keynote Speaker:
        Jeannie Lee, Massachusetts General Hospital

        Topics and Speakers:
        Non-Coding RNAs
        Art Krieg, RaNA Therapeutics

        Modulation of RNA Splicing
        Ryszard Kole, Sarepta Therapeutics
        Giles Campion, Prosensa, The Netherlands
        Stuart Peltz, PTC Therapeutics

        Immunomodulatory Oligos/RNAs
        Sudhir Agrawal, IDERA Pharmaceuticals
        Eli Gilboa, University of Miami

        RNA-mediated Reprogramming
        Victor Dzau, Duke University
        John Cooke, Stanford University

        Larry Gold, SomaLogic, Inc.
        Chris Rusconi, Regado Bioscience

        Delivery of siRNA/Antisense/Antagomirs
        Frank Bennett, Isis Pharmaceuticals
        Laura Sepp-Lorenzino, Merck

        Abstracts are welcomed on all scientific topics related to the development of RNA and other oligonucleotide-based therapeutic approaches. They should contain only new and unpublished material and must be submitted electronically by the abstract deadline. Selection of material for oral and poster presentation will be made by the organizers and individual session chairs. Status (talk/poster) of abstracts will be posted on our web site as soon as decisions have been made by the organizers.

        We are eager to have as many young people as possible to attend since they are likely to benefit most from this meeting. We have applied for funds from industry to partially support graduate students and postdocs. Apply in writing to Maureen Morrow stating need for financial support - preference is given to those submitting abstracts.

        We look forward to seeing you at Cold Spring Harbor in April.

      • Yo Simp! From over here in Perth, West Oz, this is a no brainer.

        Clearly the ever continuing anecdotal evidence revealed under every recent comment is an overwhelmingly screaming........slam dunk.

        In your leisure, please help the board brethren understand that by jumping into nonsemsical manipulation posts like the "I'm worried' variety, they are actually propelling the noise! It's called an accessory to the crime!

        It's painful to watch from the other side of the planet. Can you like get that thru to the faithful?

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