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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. 

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.

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.

A new law that allows patients with rare diseases to participate in clinical trials without losing eligibility for public health care benefits went into effect yesterday. The bill, known as the “Improving Access to Clinical Trials Act” (IACT), was championed by the Cystic Fibrosis Foundation and signed into law in October 2010.

Legislation introduced today will help more people with cystic fibrosis, and other rare diseases, participate in clinical trials without fear of losing important benefits or health coverage. The bill is sponsored by a bipartisan group of senators and legislators.

Cystic Fibrosis Foundation Vice President of Therapeutics Development Dr. Michael Boyle testified today at a hearing in front of the House Energy and Commerce Committee's Subcommittee on Health. He urged members to support H.R. 209, the Ensuring Access to Clinical Trials Act (EACT), which would make permanent a law enabling people to participate in clinical trials without the fear of losing critical benefits.