Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
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.
Historic Time in Treatment of Rare Disease as Promising Drugs Reach Phase 3 Trials
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
Meb Keflezighi, one of the world's best runners, joined Lee University and ran in the 65 Roses 5K in Cleveland, Tenn. to support the fight against cystic fibrosis. Lee University was thrilled to welcome him as part of its longstanding tradition of raising funds in support of the CF Foundation's mission.
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