Ever wondered what it takes for a potential cystic fibrosis drug to become approved by the U.S. Food and Drug Administration? Here's an explanation of the four phases of clinical research.
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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.
The radio stations are filled with holiday music, but as 2015 comes to a close, I find myself listening to an entirely different playlist. In my mind, there are not only songs that make me reflect on the path that my family has so carefully traveled so far, but also the hope and the promise of all the good things yet to come.
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This medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually benefit more than 90 percent of people with CF.
Although I'm glad that my two children with cystic fibrosis will soon have the opportunity to try Trikafta, I am also a little worried about whether they will have side effects and how well they will do on the drug long-term.
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.