SPX-101, designed to help clear mucus from the lungs of people with cystic fibrosis, has the potential to treat all CF patients — regardless of disease-causing mutations. The experimental peptide, which blocks the overactive sodium channels that line CF lung cells so more fluids are retained on airway surfaces to hydrate mucus,…
News
The Adult Cystic Fibrosis Program at West Virginia University Medicine’s Mountain State Cystic Fibrosis Center was recently awarded accreditation from the Cystic Fibrosis Foundation (CFF) for its work in the cystic fibrosis (CF) community. The accreditation is granted to institutions after a thorough application process that includes a site visit, interviews…
Some 318,000 health-related software apps are now on the market, with another 200 appearing every day. Yet a British cystic fibrosis expert questions how effective they are at managing lung disease, for reasons that range from the needless anxiety they can cause to sporadic use and concerns with patient data protection.
Vertex Pharmaceuticals employees have raised more million $1 million using the Vertex Foundation‘s matching gift program in a show of commitment to causes that include the cystic fibrosis (CF) community, a company press release states. The dollar-for-dollar matching gift program is being run through the nonprofit Vertex Foundation, established by the company…
Vertex Pharmaceuticals’ treatments for cystic fibrosis (CF) all provide considerable benefits, but their pricing is not transparent and does not align with the medications clinical value, a review of the Institute for Clinical and Economic Review (ICER)’s report by the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC)…
New research provides insights into how bacteria “talk” to each other in lung infections, which could provide strategies to block these signals and ease bacterial virulence in patients with cystic fibrosis (CF). The study, “Spatial determinants of quorum signaling in a Pseudomonas aeruginosa infection model,” was…
Combining the approved multiple sclerosis treatment glatiramer acetate with the antibiotic tobramycin improved its ability to kill antibiotic-resistant strains of Pseudomonas aeruginosa isolated from cystic fibrosis (CF) patients, new data show. These results were shared in the presentation “The repurposed multiple sclerosis drug, glatiramer acetate, is an antibiotic resistance breaker in Pseudomonas aeruginosa…
Europe’s largest annual gathering of cystic fibrosis (CF) experts ended June 9, capping four days of speeches, workshops, and poster presentations that attracted 1,832 participants from 56 countries. The largest contingents at the 41st European Cystic Fibrosis Conference, held in Belgrade, Serbia, came from the United Kingdom (304) and Serbia itself…
#ECFS2018 – Preclinical Data Shows Potential of ELX-02 to Reverse Effects of CFTR Nonsense Mutations
Preclinical studies show that Eloxx Pharmaceuticals’ lead therapy candidate ELX-02 can reverse the effects of CFTR gene nonsense mutations in mice and cystic fibrosis (CF) patient-derived organoids. These findings further support the clinical development of ELX-02 as a treatment for CF patients with CFTR nonsense mutations, which represent…
The European Cystic Fibrosis Society presented three scientists — one Portuguese, one Mexican, and one German — with the organization’s 2018 “Young Investigators Awards” on June 9, the last day of its 41st European Cystic Fibrosis Conference (ECFS) in Belgrade, Serbia. The winners are Iris Silva, Samuel Lara Reyna, and…
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