The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
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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.
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.
Today, the Cystic Fibrosis Foundation announced that it has awarded up to $400,000 to Life Edit Therapeutics Inc. to explore the application of their unique gene editing technology in CF.
Today, the Cystic Fibrosis Foundation announced that it will invest up to $8.4 million in SpliSense's Series B funding round to develop an antisense oligonucleotide therapy for people with cystic fibrosis who have splicing mutations and potentially other rare mutations.
Funding will support improved detection, diagnosis, prevention, and treatment of infections related to cystic fibrosis.
New funding will support clinical development of two novel therapies to address complications of cystic fibrosis
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.