Based in Lexington, Mass., the Cystic Fibrosis Foundation Therapeutics Lab conducts groundbreaking CF research to help rapidly advance new therapies.
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Cystic fibrosis occurs when the cystic fibrosis transmembrane conductance regulator (CFTR) protein is either not made correctly, or not made at all. By understanding how the protein is made, scientists have been able to develop treatments that target the protein and restore its function.
Two-thirds of people with cystic fibrosis in the U.S. experience debt, food insecurity, or housing issues.
The journey to end cystic fibrosis isn't a straight line. It is an evolving map with many paths and unique challenges. It requires an ambitious research agenda to accelerate treatments and drug development for the underlying cause of the disease and ultimately deliver a cure.
When I was in college, I wanted to do it all and my health wasn’t as much of a priority anymore. I learned to accept that things will not always go according to plan and to integrate routine in my life to ensure my health comes first.
Results show that people on Trikafta® enrolled in the six-week study were able to safely stop taking one of the two common CF medications without negatively affecting their health.
When I went off to college it was up to me to manage my cystic fibrosis for the first time without help from my family. I felt fine after skipping my medications and decided to stop using my noisy vest because it embarrassed me, and I wanted to make friends at my new school. As a result, I stayed in the hospital for a few weeks and left with a new perspective on life.
The Foundation and Bakar Labs will support AVECRIS Pte Ltd and Nosis Biological Sciences as they pursue genetic therapies for cystic fibrosis with their novel technologies.
The Cystic Fibrosis Foundation participated in a Congressional briefing hosted by Friends of Cancer Research on July 24 to educate members of the U.S. Senate about new processes developed by the FDA to speed the approval of breakthrough therapies.
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