The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
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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.
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
Potential treatment from Calithera Biosciences minimizes growth of germs in the lungs
Nonprofit commits $20 million to fund projects and accelerate genetic therapies for CF