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Sarepta Therapeutics, Inc. Message Board

ipsosfund1 3 posts  |  Last Activity: Jul 10, 2014 9:52 PM Member since: Mar 31, 2012
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  • Think about it.... The kids are sick of doing this test!!
    They have been patten in the back for 3 years about how good they r walking. Just like any celebrity, you start to get complacent. In their minds they know the drug works so why should they keep walking.... They just like lebron James are complacent of winning and then slack off, ie lebron is 2/4 in championships. Effort not always there.

    6mwt is effort dependent. Kids sick of the test. The differences from last data are within a confidence interval.... I say data was great in that they r all still walking!!

  • The FDA also cited the failure of PTC Therapeutics’s Ataluren trial as another example of the disconnect between levels of dystrophin and clinic outcomes. Ataluren has been selected to induce significant read-through of nonsense mutations of DMD gene, resulting in restoration of dystrophin expression in patients carrying such mutations. However, the screening assay method initially used to identify the specificity of PTC124 has been contradicted by more recent study.12 Furthermore, studies to validate the effect of PTC124 in vitro did not demonstrate any significant read-through of nonsense mutations.13 More importantly, clinical trials of Ataluren have shown only the phase IIa results with little evidence of dystrophin restoration by IHC with a very subjective scoring method.14 Unfortunately, the phase IIb muscle biopsy results have not been presented and reported to the public. Therefore, the disappointing functional data from the Ataluren and Drisapersen trials are, in fact, entirely consistent with the failure of either agent to induce production of significant amounts of dystrophin protein and do not support the notion of the “disconnect” proclaimed in the FDA report.

  • PPMD pumping and dumping shares on mom and pop
    Get out of this name and do not buy it!!

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