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Sarepta Therapeutics, Inc. Message Board

  • simp08801 simp08801 Oct 6, 2013 12:13 PM Flag

    4th biopsy issue

    I believe there are things going on behind the scene

    I believe there is a ZERO percent chance all 12 boys in the trial will have a 4th biopsy, and that belief has very little to do with the fact that each boy is a dmd boy

    not every human being responds identically to anesthesia and surgery

    some are good, some are much worse than good

    each of these boys have already had 3 biopsies over a relatively short period of time, so their parents have a pretty good idea of what happens to them when they have anesthesia and surgery

    is there a possibility that some (not all) might have it and that this would make the FDA happy? maybe

    IMO, the FDA opened its mouth and asked for something without knowing all the relevant facts

    but this would not be the first time the FDA has done something that seem ignorant of relevant facts

    we just have to hope that in this instance, its the last ...

    do not write a 4th biopsy down in non-erasable magic marker

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    • I had an open biopsy 5 weeks ago. The wound hasn't completely healed yet. The procedure weakened my left leg to the extent that it was hard to get up and down stairs for 2 weeks. I nearly fell down a flight of stairs trying to carry a 5 gallon bucket of paint 3 days after the procedure. The internal sutures haven't reabsorbed yet.

      But if it was me, I'd volunteer for another test since it might help move the program forward. To whom much is given, much is expected.

      • 1 Reply to musherga
      • What an #$%$ - these kids have "given" plenty - 2 or 3 biopsies a piece,weekly infusions, countless blood tests, poked and prodded for two years with all the time away from their families. The trial design was approved by the FDA and the boys have met the trial requirements. Even IF another biopsy would provide some critical information, it would still be wrong for the FDA to request it. The fact that it is not likely to provide any meaningful data than already exists makes it unethical. Maybe if you had fallen down the stairs it might have knocked some sense into you!

    • simp, your passion is very timely. This is an extremely sensitive and dramatic situation. Kids that coming out of death should-could-may help or ignore ( the fear of pain) the desperate call of other boys, who are doomed to die without an access to eteplirsen.

    • see this quote:

      " parents been asked to consider a 4th biopsy. anesthesia always a risk with these kids, open muscle surgery- recovery varied."

      key words: RECOVERY VARIED

      IMO, its TOO varied to assume all would do it

      • 2 Replies to simp08801
      • Based on the severety of the disease, the lack of any other effective drugs, and the remarkable and statistically significant data over almost 2 years, I have to think the FDA has all that it needs to approve for exon 51. If the 4th biopsy has more to do with the other exons, and establishing dystrophin as an acceptable surrogate marker towards some sort of plaftorm approval, (LIKE THEY ALREADY DID IN EUROPE - hint, hint FDA), then hopefully data from less than all 12 boys will be acceptable. towards that end. It does seem inhumane to request yet another muscle biopsy solely to support approval of Etep/exon 51. Maybe the parents should all agree to put their already suffering kids through another biopsy, once each and every member of the FDA has had one too?

      • True, however, you only need a few not all.

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