News

A new agreement will give people in Scotland with cystic fibrosis access to the CF combination treatments Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) through the country’s health system, according to Vertex Pharmaceuticals, the company that sells both medicines. The announcement follows a prior rejection by the …

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…

How well Acceptance and Commitment Therapy (ACT), or tailored “talk” therapy that can be delivered via telemedicine, helps people with cystic fibrosis (CF) cope with mental health issues will be the focus of an upcoming study. Supported by a three-year, $960,000 grant from the Boomer Esiason Foundation, the study is…

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…

Claire’s Place Foundation is preparing for its 5th Annual Glow Ride event to raise money to support families affected by cystic fibrosis. The 3.4 mile-long bike ride will take place Saturday at 5:30 p.m. at the piers of Hermosa Beach and Manhattan Beach in California. In addition…

Blocking a pro-inflammatory protein complex, broadly known as the neutrophil inflammasome, represents a new way of treating chronic Pseudomonas aeruginosa infections in cystic fibrosis (CF) patients, a proof-of-concept study demonstrated.  The study, “Specific Inhibition of the…

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,…

Clinical centers with high screening rates for cystic fibrosis-related diabetes (CFRD) were more likely to achieve early CFRD diagnosis and prevent lung function decline, a study has found. This was reported in a study, “The association of pediatric cystic fibrosis-related…