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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Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments
I’ve learned that paying too much attention to the idealized lives of people on social media can give me a skewed vision of my own.
At age 5, my daughter started to feel the traumatic emotional effects of cystic fibrosis. Fortunately, the care center’s social worker helped her find a healthy path through it.
I wish people knew that even if I don’t look sick, I still struggle with the mental and physical aspects of cystic fibrosis. Even my family and friends don’t truly understand what this disease does to your mental health.