Skip to content
  • Categories
  • Recent
  • Tags
  • Popular
  • Users
  • Groups
Skins
  • Light
  • Cerulean
  • Cosmo
  • Flatly
  • Journal
  • Litera
  • Lumen
  • Lux
  • Materia
  • Minty
  • Morph
  • Pulse
  • Sandstone
  • Simplex
  • Sketchy
  • Spacelab
  • United
  • Yeti
  • Zephyr
  • Dark
  • Cyborg
  • Darkly
  • Quartz
  • Slate
  • Solar
  • Superhero
  • Vapor

  • Default (No Skin)
  • No Skin
Collapse

The New Coffee Room

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

Cystic fibrosis drug

Scheduled Pinned Locked Moved General Discussion
4 Posts 2 Posters 35 Views 1 Watching
  • Oldest to Newest
  • Newest to Oldest
  • Most Votes
Reply
  • Reply as topic
Log in to reply
This topic has been deleted. Only users with topic management privileges can see it.
  • 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/

    "The intelligent man who is proud of his intelligence is like the condemned man who is proud of his large cell." Simone Weil

    1 Reply Last reply
    • jon-nycJ Online
      jon-nycJ Online
      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.

      Thank you for your attention to this matter.

      1 Reply Last reply
      • MikM Offline
        MikM Offline
        Mik
        wrote on last edited by
        #3

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

        "The intelligent man who is proud of his intelligence is like the condemned man who is proud of his large cell." Simone Weil

        1 Reply Last reply
        • jon-nycJ Online
          jon-nycJ Online
          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.

          Thank you for your attention to this matter.

          1 Reply Last reply
          Reply
          • Reply as topic
          Log in to reply
          • Oldest to Newest
          • Newest to Oldest
          • Most Votes


          • Login

          • Don't have an account? Register

          • Login or register to search.
          • First post
            Last post
          0
          • Categories
          • Recent
          • Tags
          • Popular
          • Users
          • Groups