Phase 2 clinical trial results were announced today for a potential next-generation modulator to treat people with two copies of the most common cystic fibrosis mutation, F508del.
Site Search
A new drug intended to make it easier to clear mucus from the lungs of people with cystic fibrosis showed promising results in a Phase 2 clinical trial. The drug, called SPX-101, blocks the overactive sodium channels that line CF lung cells.
Today Vertex Pharmaceuticals Inc. announced the two next-generation CFTR modulator candidates that will progress into Phase 3 clinical trials.
Cystic Fibrosis Foundation Drug Development Model Spawns More Than 30 Promising Therapies
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.
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.