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Thread: Testing CFTR Modulation drugs like Kalydeco, in vitro, on rare mutations.

  1. #1
    Junior Member
    Join Date
    Sep 2016
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    18

    Testing CFTR Modulation drugs like Kalydeco, in vitro, on rare mutations.

    A lab in the Netherlands have found a new, reliable way to test drugs like Kalydeco in vitro, by growing an organoid of a patientīs intestine. If the gut reacts positively on the drug this could be a way to help patients with rare mutations (with residual function) to access drugs like Kalydeco even though these mutations havenīt been tested in clinical trials (and hence havenīt been included in the approval by the FDA).
    The primary aim seems to be to improve in vitro studies, making them more reliable and applicable on rarer mutations and I donīt know if it will be used as means for physicians to prescribe experimental drugs to patients who would otherwise be left out. I just hope cf-physisians will see the potential for clinical practice here!

    http://www.sciencedirect.com/science...34590916302958

  2. #2
    Super Moderator
    Join Date
    Sep 2016
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    126
    Wow! Thanks for sharing. This sounds so promising.

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