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CFF-Championed Bill Eliminates Barriers to Participation in Clinical Trials for People with CF and other Rare Diseases

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

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

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.