PER percheron therapeutics limited

Ann: WHO selects proposed INN for ATL1102, page-11

  1. 14,651 Posts.
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    https://www.biospace.com/article/as-sarepta-s-elevidys-closes-in-on-full-approval-experts-weigh-in

    With the deaths of young DMD boys and the long term side effects in question.. I can’t see the FDA passing the gene therapy drug .
    Not only that the high cost and the drug failed to show efficacy
    Imo
    the FDA will not give approval for the entire DMD population

    PER is only days away from full enrolment ..
    Then the countdown begins…
    We already have more than 10 boys who are dosing beyond the 6 month period
    All good.
    Also the Tox study is completed or near enough
    the fact that they haven’t stoped the trial tells me that the 9 month study is successful.

    ..
 
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