Small Steps and Giant Leaps

For the first time ever, I am posting on the fly.  So, pardon the typos and I’ll keep it brief.

Today was a tremendous day for the cystic fibrosis (CF) community.  Today, Vertex Pharmaceuticals Inc. announced the FDA’s approval of KALYDECO, the first drug to treat the underlying cause of cystic fibrosis.  This is groundbreaking – not just for those affected by CF, but for the world of medicine!  The benefits of this collaboration between Vertex and the CF Foundation will have a tremendous ripple effect.

We in the CF community have watched Kalydeco, formally known as VX-770, progress through the CFF’s research pipeline for many years now.  This progress, each small step, was made possible by the dedication and financial backing of patients, families, friends, clinicians, researchers, volunteers, individual donors, corporations and CFF staff.  The CFF receives no federal funding, and the major drug companies are not interested in funding CF research because, for them, not enough people have the disease to make it a profitable endeavor. The CFF must fund its own research—and they are a national model in doing so!

Limitless thanks to Vertex Pharmaceuticals for making an investment in this orphan disease, and to every single person who has given time, talent and/or treasure to this cause.

To those have wondered why I’ve continued my brother’s fight against CF when I “could’ve put CF behind me”, let today’s news serve as your answer.  NEVER give up the good fight!  NEVER give up hope!  Today, a giant leap was made towards the cure for cystic fibrosis!  Today, a big piece of Andy’s dream – our dream, was realized!

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7 Comments

  1. It is a huge step, you are so right. Thanks for sharing this!

    Reply
  2. Hi Blake, I just read the story of your connection to CF through your brother. I am sorry that you had to live with CF and see the worst outcomes possible.

    Having said that, I admire your spirit! And today is quite the day to celebrate, right? 🙂

    Reply
    • Hey Suzanne! I was thrilled to find you on WordPress today. CF SUCKS – and you know that better than I do! I am sorry for all you and your brother have to endure.

      Today IS a quite a day to celebrate, though. It makes me all the more excited about the VX-770 / VX-809 combo research.

      Love your blog! Looking forward to following. 🙂

      Reply
  3. Blake I enjoyed reading your blog. CF sucks beyond what I can express in words. I feel grateful to be alive but I also feel guilty for being alive when so many others have not survived this horrible disease. I wish I could bring everyone that has given their life to this disease back for a second chance. With the announcement this week maybe everyone would get the second chance they deserve. I wish I had the right words to say but I don’t. Keep writing!

    Reply
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