Historic Time in Treatment of Rare Disease as Promising Drugs Reach Phase 3 Trials
Site Search
This month, 15-year-old Molly Bonnell and her sister Emily, 13, who have cystic fibrosis, discovered how easy it is to make their voices heard in Congress -- without leaving their living room.
Legislation to Help Speed Research for Cystic Fibrosis and Other Rare Diseases
Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
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.
PTC Therapeutics has discontinued development of ataluren as a potential treatment for people with cystic fibrosis caused by a nonsense mutation. The Cystic Fibrosis Foundation is supporting efforts to pursue treatments for people with CF who have a nonsense, splicing or other rare mutations.
Bill Would Remove Financial Penalties for Participating in Research Studies
The Cystic Fibrosis Foundation today mourns the passing of legendary sports journalist Frank Deford, whose young daughter Alex lost her fight to CF in 1980, and who served as chair of the Cystic Fibrosis Foundation's Board of Trustees from 1982-1999.