Children in the U.S. with cystic fibrosis (CF) tend to have better lung function than young people with the disease in the U.K., a new study has found, with researchers saying the disparity is likely due to differences in treatment between the two countries. In the U.S., children are more…
Regulatory agencies in the European Union and in the U.K agreed to consider expanding the use of Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to children with cystic fibrosis (CF) who have at least one F508del mutation starting at age 6. The European Medicines Agency (EMA) and the U.K.’s Medicines…
Vertex Pharmaceuticals has made a $32 million purchase from Concert Pharmaceuticals related to future milestones from the 2017 agreement when Vertex purchased Concert’s investigational treatment for cystic fibrosis (CF), VX-561. According to Roger Tung, PhD, Concert’s president and CEO, the purchase will help Concert as it continues…
SpliSense has raised $28.5 million in funding to advance its antisense oligonucleotide (ASO) platform for the treatment of cystic fibrosis (CF) caused by certain rare mutations, and of other genetic lung diseases. ASOs are molecules designed to target the messenger RNA (mRNA), the intermediate molecule derived from DNA that guides protein production. Money…
A new biomaterial made up of nanoparticles showed a potential to improve — by up to 100,000 times — the efficiency of tobramycin, an antibiotic used to treat cystic fibrosis (CF) patients with Pseudomonas aeruginosa infections. Tobramycin, carried within a nanostructured material, completely eradicated P. aeruginosa infection in a…
Vitamin D intoxication may occur in people with cystic fibrosis (CF) due to accidental errors made by pharmacists in preparing prescriptions, a study has found. Indeed, some 13 patients in the study were taking a dose of vitamin D that was nearly 70 times higher than the prescribed one,…
Combining AzurRx BioPharma‘s investigational therapy MS1819 with standard pancreatic enzyme replacement therapy (PERT) can improve fat absorption in cystic fibrosis (CF) patients with severe exocrine pancreatic insufficiency (EPI), interim data from a Phase 2 clinical trial indicate. “The overarching goal of our MS1819 program is to provide a safe and…
A review of 57 real-world studies found that people with cystic fibrosis (CF) who take Kalydeco (ivacaftor) experience improvements in lung function, nutrition, and quality of life (QoL). Reduced healthcare system use, lower Pseudomonas…
Long-term use of Orkambi (lumacaftor/ivacaftor), an approved cystic fibrosis (CF) treatment, is safe and effective for children between ages 2 and 5 with two copies of the disease-causing F508del mutation in the CFTR gene, a study reported. It draws on results from a Phase 3 open-label extension trial…
When it comes to raising funds for cystic fibrosis (CF), the Cystic Fibrosis Foundation is committed to relentlessly pursuing research initiatives until “CF stands for cure found.” Last year, the foundation put a total of $218.1 million toward research and spent $80 million on its…
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