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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The only thing that is certain in my life is the uncertainty of my teenage daughter's next hospital stay. Despite this, she has somehow managed to supervise her own academic life from a hospital room.
Looking back at my freshman year, I realize that putting college before my CF wasn't the first thing I had all wrong.
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.
The following questions and answers address concerns from the community about COVID-19 and school.
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