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.
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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.
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Choosing to volunteer in a
When Brady was diagnosed with CF, I felt like I'd been thrown into a river without a paddle. But then I realized my “paddle” was CF advocacy, and it could help steer our boat in the right direction.
There have never been as many new CF drugs in development as there are today. In the second plenary at NACFC, Dr. George Retsch-Bogart outlined the progress we've made, the road ahead and the changes needed to make it all possible. Read on for my key takeaways.
As Congress weighs budget appropriations, we look at long-term funding for the National Institutes of Health. We decided to sit down with the Foundation's senior vice president of research affairs to learn more about why the work being done at NIH is so important in the search for a cure for CF and other serious, rare diseases.
Last week I travelled to D.C. to serve on a panel discussing the recent developments in precision medicine. I've got to say, it was pretty neat.
Clinical trials that test potential drugs and therapies in people with cystic fibrosis are a major part of CF research. They take place at Cystic Fibrosis Foundation-accredited care centers all over the United States and enroll people with CF of all ages.
Robust funding for the National Institutes of Health helps ensure innovation in basic research and a full pipeline of cystic fibrosis therapies. A well-resourced U.S. Food and Drug Administration helps advance therapies that are safe and effective.