Today Vertex Pharmaceuticals Inc. announced the two next-generation CFTR modulator candidates that will progress into Phase 3 clinical trials.
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The Cystic Fibrosis Foundation's Greater New York Chapter recently recognized leading journalist Scott Pelley at the 14th annual Breath of Life Gala for his unwavering dedication to those living with CF.
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.
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 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.
The studies aim to improve early detection of chronic lung allograft dysfunction (CLAD), the leading cause of lung transplant failures.
New funding awards include up to $2.6M to Eloxx Pharmaceuticals to identify potential therapies for CF nonsense mutations
Foundation to fund up to $110 million for collaboration to accelerate the development of genetic therapies for CF