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.
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.
The top priority in clinical research is protecting the safety of people who participate. Learn more about the numerous layers of review that help keep participants safe.
When my 8-year-old daughter with CF found out that the disease is potentially fatal, I was grateful for her optimistic views on life and death. But deep down, I was saddened with the thought of a cure being so far away that I wouldn't be around to see it. Today, my fears have dwindled.
Dr. John P. Clancy shares new developments in drugs that restore airway surface liquid in the lungs of people with cystic fibrosis, making it easier to clear mucus.
Dr. James Chmiel shares new developments in drugs to reduce inflammation in the lungs and help prevent lung damage.
Dr. Jennifer Taylor-Cousar shares new developments in drugs that fight infections in the lungs of people with cystic fibrosis.
For the first time, the Cystic Fibrosis Foundation invited non-CF experts to its annual research conference to meet with CF researchers. The conference -- New Technologies Advancing Toward a One-Time Cure -- in Savannah last month focused on the challenges being faced in gene editing, gene delivery and stem-cell biology and laid the foundation for new collaborations.
Drs. Drucy Borowitz and Manu Jain share new developments in drugs that will treat the underlying cause of cystic fibrosis.