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Single dosing of orally inhaled POL6014, an experimental therapy to treat chronic lung inflammation, can effectively block the activity of a pro-inflammatory enzyme in the lungs of people with cystic fibrosis (CF), results from a Phase 1 clinical trial show. The data were published…

The clinical effectiveness and cost-benefit of several cystic fibrosis (CF) therapies will be the focus of a final evidence report by the Institute for Clinical and Economic Review (ICER), which is currently seeking feedback from CF stakeholders for the report. The report will mainly evaluate the effects of…

Through a new partnership with the Cystic Fibrosis Foundation (CFF), California-based winery Tarpon Cellars will donate $1 to the organization’s Georgia chapter for each bottle of wine sold. The funds will help support research and treatment development for cystic fibrosis (CF), an inheritable…

Amikacin liposome inhalation suspension (ALIS) showed the same efficacy and similar safety profile as tobramycin inhalation solution (TIS) for the treatment of cystic fibrosis (CF) patients with Pseudomonas aeruginosa lung infection, a comparative clinical study showed. The study, “Amikacin liposome inhalation suspension for chronic Pseudomonas aeruginosa infection in cystic fibrosis,” was…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

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