Eloxx Pharmaceuticals announced it expects to share data later this year from the first four treatment groups of a Phase 2 program evaluating the investigational therapy ELX-02 in people with cystic fibrosis (CF) who have at least one G542X mutation. “Given the substantial efforts of our clinical team,…
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Functional residual capacity (FRC), a measure of the volume of air left in the lungs at the end of a normal exhalation, may help detect lung disease in children diagnosed with cystic fibrosis (CF) via newborn screening, a study suggests. Also, a cut-off value of 36 breaths per minute may…
Arrowhead Pharmaceuticals has voluntarily paused new screening, enrollment, and dosing in its Phase 1/2 trial of ARO-ENaC, an investigational treatment for cystic fibrosis (CF). The decision came after unexpected signs of local lung inflammation occurred in an ongoing chronic toxicology study taking place in rats. “The safety of…
Cystic fibrosis (CF) patients with specific variants in the Tensin 1 gene may be at risk of low body mass index (BMI), a measure of nutritional status, if they carry the common CF-causing F508del mutation in both copies of the CFTR gene, a study reports. This makes Tensin 1 (TNS1) a…
Cystic fibrosis (CF) patients with lower aerobic exercise capacity — assessed with a non-invasive cardiopulmonary exercise test — have a 4.5 times higher risk of future pulmonary exacerbations, according to a small 10-year study in Greece. Aerobic capacity, or VO2peak, is the maximal amount of oxygen a person’s…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
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…
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