Researchers are exploring treatments that will keep people with cystic fibrosis as healthy as possible until a cure is found. In the first plenary at the NACFC, two CF scientists explain the progress of current research.
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For those of us without a medication that targets our cystic fibrosis mutation, it's important to remember that our life with CF is a marathon.
Cystic Fibrosis Foundation Therapeutics Inc. made its first major award last month to advance gene editing research in cystic fibrosis. Editas Medicine Inc. will explore the potential of using what some scientists refer to as “a tiny pair of scissors” to snip out the genetic defect that causes CF.
The Cystic Fibrosis Foundation is funding research into gene editing techniques to see if they can be used to fix the mutations that cause cystic fibrosis. One of the most popular techniques is CRISPR-Cas9. To see how this might work for CF, watch this video.
The CF Foundation is focused on bringing forward new therapies to help people with CF enjoy the best health and quality of life.
As the mother of a child with CF and volunteer for the CF Foundation, I know first-hand the extraordinary things that Dr. Bob Beall has done for our community, and I am grateful for the legacy of hope that he leaves behind.
Earlier this month, we learned that the new CF drug Orkambi was approved by the FDA. This was such an exciting, memorable and emotional day for everyone in the CF community. We heard from many who were thrilled about the potential for this drug to help people with CF and also heard from those who would not benefit from this breakthrough. Following are some reactions from those this impacts the most: people with CF and their loved ones.
I've lived my whole life like it has an early expiration date. Recently, a drug was FDA-approved that could change that. I'm grateful, but I'm also scared…
Although I had been told that my coughing would stop, I wish I had known more about the transformative change that Trikafta® would have on my life. It has almost made me wistful for the time when I was sick, back when I was more in tune with what my body was experiencing.
Now that I am feeling healthy on Trikafta®, I no longer feel special for thriving despite the challenges of cystic fibrosis. I don’t feel like I belong in the CF community anymore, and I miss that sense of camaraderie that comes from a shared struggle.