A combination of bacteria-infecting viruses and antibiotics can be used to effectively protect lung cells against infection by Pseudomonas aeruginosa, a bacteria that commonly causes lung infections in people with cystic fibrosis (CF), according to a study led by researchers at the University of Geneva (UNIGE). The model…
News
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
The nonprofit Cystic Fibrosis Canada has teamed up with technology company iMD Health Global (iMD) to make user-friendly digital health tools for cystic fibrosis (CF) available to both patients and healthcare professionals. The  goal is to boost understanding of the disease and how to best manage it. Through…
A first-of-its-kind, observational study will allow patients with cystic fibrosis (CF) to use their smartphones to monitor the ways in which Trikafta (elexacaftor/tezacaftor/ivacaftor) affects their daily lives. Sponsored by the Cystic Fibrosis Foundation, the now-enrolling HERO-2 study (NCT04798014) will collect patients’ self-reported feedback for one year…
The therapeutic benefits of the triple-combination medication Trikafta are mostly due to the effects of elexacaftor, a next-generation corrector of CFTR, the faulty protein in cystic fibrosis (CF), a study has found. These findings suggest that new combinations with elexacaftor may be even more effective than Trikafta. Trikafta,…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…
Treatment with Orkambi (ivacaftor/lumacaftor) led to significant improvements in lung health, particularly related to clearing mucus, among people with cystic fibrosis (CF), according to a real-world study of its use in France. The investigation provided a first high-resolution CT scan-based assessment of the medication’s effectiveness, and results also indicated…
Physical activity, undertaken regularly over the long term, appears to complement other therapies in managing cystic fibrosis (CF), a study in a group of active and non-active patients found. Its results also showed that tumor necrosis factor alpha (TNF-alpha) may serve as a biomarker of inflammation to monitor the…
As part of its Path to a Cure initiative, the Cystic Fibrosis Foundation announced three new awards to fund research projects that seek to advance the treatment of cystic fibrosis (CF). The largest award, of up to $2.6 million, was given to Eloxx Pharmaceuticals to aid in the…
The legacy of Mallory Smith, who died nearly four years ago from a cystic fibrosis-related superbug infection in her lungs, is living on in the form of raising awareness and funds for a novel treatment called phage therapy that might have saved her life. Bacteriophages, or simply phages,…
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