News

Vertex Pharmaceuticals has reached reimbursement agreements with health authorities in France and Italy for its various cystic fibrosis (CF) treatments, including Kaftrio (ivacaftor/tezacaftor/elexacaftor), a next-generation therapy approved for use with ivacaftor. With these agreements — which bring the treatments into each country’s public health program — about 3,000 CF patients…

Symdeko (tezacaftor/ivacaftor) favors weight gain, shortens hospital stays, and reduces the number of days on antibiotics in patients with cystic fibrosis (CF), a real-world study of its use at a Scottish clinic reported. However, no significant benefits in patients’ lung function were seen over about a year of…

Kalydeco (ivacaftor) safely and effectively improved lung function, growth, and nutrition in children and adults with cystic fibrosis (CF), while reducing rates of pulmonary flares and hospitalizations, according to a real-life study in France. These findings were consistent with those reported in previous clinical trials and real-world…

Chronic Pseudomonas aeruginosa infection in adults with cystic fibrosis increases their likelihood of developing CF arthropathy, a painful inflammation of the joints, a German study reported. Additional risk factors for CF arthropathy include older age, female sex, and more severe disease. These findings were published in the Journal of Cystic Fibrosis, in…

Reduced levels of the VIP hormone in the pancreas due to low nerve supply may contribute to the development of early diabetes in people with cystic fibrosis (CF), a study in mice suggests. The study, “VIP reduction in the pancreas of F508del homozygous CF mice and early signs…

Cystic fibrosis (CF) carriers — and especially men with mutations in only one copy of CFTR, the gene associated with CF — are at a higher risk of developing a severe COVID-19 infection, one that carried a greater need for ventilation and could be quickly fatal, according to…

Health Canada has approved Trikafta  as a triple-combination therapy for people with cystic fibrosis (CF), ages 12 and up, who have at least one F508del mutation, the most common CF-causing mutation. This decision is expected to give about 1,100 people access for a first time to a therapy that directly…

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

A new strategy to correct so-called splicing mutations could help deliver nucleic acid therapies called oligonucleotides to cells in the lung, a new study suggests. This approach may aid in the development of new treatments for people with cystic fibrosis (CF), according to researchers. “With our oligonucleotide delivery platform,…

A mobile, dietary self-monitoring app used for six months to track food choices and manage pancreatic enzyme replacement therapy (PERT) helped children with cystic fibrosis (CF) to better meet disease-specific nutritional guidelines, a study shows. Still, the nutritional changes made were not sufficient to reach the standards of…