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There's an interesting discussion on gene therapy trials and the...

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    There's an interesting discussion on gene therapy trials and the implications for Daybue.
    There seems to be uncertainty about the implications of being on Daybue.

    "There are two different types of GT trials going on right now. Neurogene & Taysha are the companies. For Taysha there is no Daybue exclusion from what I gather. With Neurogene, it’s a different story. They don’t want any patients who have ever been on Trofinetide or Daybue."

    "Exclusion criteria is likely to change over time and depending on the type of therapy circumstances. But initially, it sounds like Neurogene doesn’t want any other treatments or trial drugs interfering with their work."

    "They are having people do a wash out period if they are doing a gene trial. So for anyone who is taking DAYBUE and qualifies for the gene trial they will give you the clinical guidelines on how and what to do…...it’s a 12 week break before starting a gene therapy trial"

    "My doc said that they won't take them for gene therapy if they have taken daybue! Correct me if I'm wrong but that's what our rett clinic neuro told me”


    Very interesting scenario. Some may want to stop Daybue and trial the Gene Therapy. You’d think most likely the ones who didn’t have great results or had too significant side effects.
    However it would be hard to stop if things were going well. As a company doing trials I’d be very hesitant to take someone who was on Daybue, even after being off for 12 weeks.
    You wouldn’t want to take a risk that longer term deterioration could occur once having stopped Daybue, and influencing the outcome.
    Surely patients on Daybue would not be allowed.

    And finally here we have the huge advantage of first in class where we don’t have to worry about finding enough patients for the trial and not having to deal with patients not wanting to stop what was already working
    Last edited by goldenchook: 24/10/23
 
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