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The New Coffee Room

  1. TNCR
  2. General Discussion
  3. Cystic fibrosis drug

Cystic fibrosis drug

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  • MikM Offline
    MikM Offline
    Mik
    wrote on last edited by Mik
    #1

    I was just watching Boomer Esiason onTv and got curious about his son who has Cystic Fibrosis. I was surprised to learn a med was approved in 2019 that has been remarkably effective at treating the protein deficiency that causes the disease.

    https://www.lovewhatmatters.com/cystic-fibrosis-illness-boomer-esiason-chronic-illness/

    “I am fond of pigs. Dogs look up to us. Cats look down on us. Pigs treat us as equals.” ~Winston S. Churchill

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    • jon-nycJ Offline
      jon-nycJ Offline
      jon-nyc
      wrote on last edited by
      #2

      Yeah, Vertex. They’re now working on my disease, also with a small molecule corrector. I know them quite well.

      "You never know what worse luck your bad luck has saved you from."
      -Cormac McCarthy

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      • MikM Offline
        MikM Offline
        Mik
        wrote on last edited by
        #3

        I thought you might. And idea when something might be approved?

        “I am fond of pigs. Dogs look up to us. Cats look down on us. Pigs treat us as equals.” ~Winston S. Churchill

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        • jon-nycJ Offline
          jon-nycJ Offline
          jon-nyc
          wrote on last edited by jon-nyc
          #4

          They had two molecules wash out but with successively better results. They’re probably 2 years away from clinical trials in their 3rd attempt.

          It took them probably 10 years to come up with Trikafta for CF. Though they had successful intermediate products.

          "You never know what worse luck your bad luck has saved you from."
          -Cormac McCarthy

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