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.
The top priority in clinical research is protecting the safety of people who participate. Learn more about the numerous layers of review that help keep participants safe.
Learn how Rachel Kinney fits studying for a computer science major, an active social calendar and daily cystic fibrosis treatments into her life.
Our next “Living Today” video features Jordan Scott, 22, whose biggest challenge involves gaining weight. But through her love of studying nutrition, she's been able to overcome her fear of the inevitable: a feeding tube.
Finding a Balance Between CF and High School