Cystic Fibrosis News Moderator

A study in cells of CF patients with F508del mutation found ARINA-1 may be better than current options for clearing mucus, especially in combo with Orkambi. Learn more here.

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A real-world study of Orkambi’s use by 827 patients, including those kept from trials, supported its broad label based on lung function and weight gains. Click here to learn more.

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Rage Biotech, building on research done at Australian universities, will focus on a protein that amplifies inflammation to treat inflammatory lung diseases. Read more about this research here.

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Mutations that cause P. aeruginosa to lose motility are linked to higher production of complex sugar molecules involved in biofilm formation, a study says. Find out more here.

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Women caring for their children with cystic fibrosis express greater anxiety related to COVID-19 than the young patients themselves, a survey found. Read the full story here.

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Kaftrio (ivacaftor/tezacaftor/elexacaftor) will be available in England as soon as the medication is approved by the European Commission. Click here to learn more.

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A survey found cystic fibrosis patients using NuvoAir’s home monitoring system made better use of telemedicine and needed fewer in-person doctor visits. Click here to read more.

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Alexion Charitable Foundation has awarded $1.1 million to NORD and Global Genes to support rare disease patients amid the COVID-19 pandemic. Read the full story here.

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Enrollment has resumed in Eloxx’s trial of ELX-02 for CF in Israel and Europe — but not in the U.S. — after being suspended amid the COVID-19 crisis. Click here to learn more.

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4D Molecular Therapeutics will receive up to $14 million from the CFF to support the development of a customized gene carrier. Click here to learn more.

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Most people in the U.K. are unaware of the challenges faced by people with CF, according to results of a poll commissioned by the Cystic Fibrosis Trust. Read the full story here.

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Exposure to second-hand smoke is linked to changes in levels of metabolites and activation of signaling pathways that enhance inflammation. Read more here.

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The Black Women’s Health Imperative has created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Read more about it here.

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The European Commission has approved Kalydeco (ivacaftor) to treat children and adolescents with CF who carry the R117H mutation in the CFTR gene. Read more here.

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Thousands of people with cystic fibrosis in England over the age of six will soon be equipped with digital home spirometry kits. Read more here.

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A study shows that the mobile app MyCyFAPP, which helps patients manage PERT regimens, can improve the quality of life of children with CF with severe GI symptom. Click here to read the full story.

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Lara Govendo believes that vulnerability and unity are key to overcoming struggles. Click here to read more from Lara.

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International Biophysics and the Colton Underwood Legacy Foundation are partnering to deliver the AffloVest to Benjamin Clackum as part of the Legacy Project. Learn more about the project here.

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Investigational treatment Encala is safe, well tolerated, and increased fat absorption in patients with CF with pancreatic insufficiency, a trial analysis shows. Click here to read more.

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Oxford Hospitals Charity has purchased and delivered machines able to measure lung function at home to 20 young people with in the U.K. Read the full story here.

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