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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.

With a record 45 clinical trials either ongoing or starting up in 2016, research coordinators and principal investigators throughout the Therapeutics Development Network took time out in San Antonio recently to discuss clinical research and put attendees through boot camp.

Sr. Vice President for Policy and Community Affairs Mary Dwight reflects on the passage of the Ensuring Access to Clinical Trials Act (EACT) and what it means for the community.

The CF Foundation is focused on bringing forward new therapies to help people with CF enjoy the best health and quality of life.