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Sarepta Therapeutics, Inc. Message Board

  • new_hunter1 new_hunter1 Oct 18, 2013 12:57 AM Flag

    If eterplirsen were effecacious, why

    at 96 wk the difference in 6mw between the treatment and delayed treatment groups was increasing rather than decreasing?


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    • You wrote: 'At these boy's ages all would be wheelchair bound if not on the drug'
      DMD has a slow onset. The disease progression also varies among patients. They do not become un-ambulatory at a narrow window. BTW, there are two boys in the treated group became un-ambulatory. The co just took them out of the tally in the 6mw. What is the justification for doing so, just because they cannot walk? The number in the 6mw for these two boys should be 0, rather than completely removed in IMHO. Just wondering how this would impact the data they have now.

      • 1 Reply to new_hunter1
      • You should pay attention to your own posts - you say that they "don't become un-ambulatory at a narrow window" and then follow up with "two boys in the treated group became un-ambulatory". The two boys that lost ambulation did so within 6 months of entering the trial, after meeting the same enrollement criteria as the rest of the boys in the study. The study was designed to enroll boys that were on the verge of losing ambulation - i.e. in a "narrow window". Based on the other posts I have read of yours, you have no knowledge of the disease, trial design or other pertinent issues. Combine that with the fact that you refute yourself in your own posts makes it unlikely that you are going to post anything intelligent - give it a rest and move on.

    • One of the kids broke their leg dumb #$%$

    • While it is interesting to read the replies of "Gosh, if you don't know, then you are an idiot" - I'd like to offer a different approach. There are still many variables that cannot be isolated including the genetic uniqueness of each patient. It cannot be shown - what the patient would have been - if they didn't take the drug - so you can't really assume a lack of efficacy either, based on a small variance in the 6mw. It could simply be an anomaly. This is one of the major problems with such a small test group. We simply don't know. However, there is still plenty of corroborating data to prove efficacy across the patient population, and that data grows every day.

    • At these boy's ages all would be wheelchair bound if not on the drug....natural history of this disease does it to all.Sarepta has some of the oldest kids in the trial currently .I think first before making comments you should study the disease .

    • Do you know how to read a Kaplan-Meier curve? Cancel drugs get approved even if there are deaths in the treatment arm. You don't understand clinical trials at all, and you are not a scientist either. Go hit the textbooks before coming here typing non-sense.

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