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