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.
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Ever wondered what it takes for a potential cystic fibrosis drug to become approved by the U.S. Food and Drug Administration? Here's an explanation of the four phases of clinical research.
Check out a post by Brandon, an adult with CF, on clinical trials and why he found one to be rewarding even though the drug wasn't ultimately approved.
Bill Would Remove Financial Penalties for Participating in Research Studies
Learning that your baby has cystic fibrosis may come as a shock, especially if you do not have a family history of the disease, but you are not alone.
As a CF mom, I'm always in a state of wonder about whether the choices I'm making are the right ones for my son with CF and the rest of the family.
CFF-Championed Bill Eliminates Barriers to Participation in Clinical Trials for People with CF and other Rare Diseases
Today, the Cystic Fibrosis Foundation announced a new award of up to $2 million to Eloxx Pharmaceuticals Inc. to support the global Phase 2 clinical program of ELX-02, a potential therapy to treat people with cystic fibrosis who have nonsense mutations.
There are many ways you can support a family with a child or children with cystic fibrosis. Educating yourself about the disease and its treatments and offering to help without being asked are some of the best ways you can show you care.