cystic fibrosis

People who carry certain genetic mutations associated with cystic fibrosis, (CF) but who do not have the disease, are at risk for some gastrointestinal problems similar to those reported by CF patients, a large-scale study shows. This finding helps better understand the impact of genetic mutations linked to CF, as…

The Cystic Fibrosis Foundation (CFF) is celebrating the United States Food and Drug Administration’s decision to approve Trikafta. The therapy — a triple combo based on elexacaftor, tezacaftor, and ivacaftor, which is being developed by Vertex Pharmaceuticals — was approved on Oct. 21 for people with cystic fibrosis (CF)…

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the use of Kalydeco (ivacaftor) in infants ages 6 months to a year with cystic fibrosis (CF). The decision was announced by Vertex Pharmaceuticals, the…

Vertex Pharmaceuticals has taken a major step toward treating 90% of all people with cystic fibrosis (CF), its long-stated goal, with the U.S. Food and Drug Administration’s approval of Trikafta (elexacaftor, tezacaftor, and ivacaftor) as a next-generation, triple combination treatment for CF. This triple combination treats people with the most…

Boehringer Ingelheim announced the enrollment of a first patient in its Phase 2 BALANCE-CF 1 trial assessing the safety and efficacy of BI-1265162 to treat cystic fibrosis (CF) regardless of a person’s disease-causing mutations. BI-1265162 is an epithelial sodium channel (ENaC) inhibitor that works to prevent cells lining…

AzurRx BioPharma has started dosing patients in a Phase 2 clinical trial assessing its investigational therapy MS1819-SD in combination with porcine enzyme replacement therapy (PERT) for people with cystic fibrosis (CF) who have severe exocrine pancreatic insufficiency (EPI). People with CF can develop EPI when…

Regular exercise over the long term improves lung function in people with cystic fibrosis (CF) by lowering levels of a pro-inflammatory hormone called adiponectin, a new study suggests. Measuring the levels of adiponectin and another hormone known as leptin could also be useful in monitoring CF progression, the…

John Pirozzi, a representative of Chelsea Financial Services, will participate in the TCS New York City Marathon to raise money for cystic fibrosis research, the company announced. Pirozzi is specifically running in support of the Boomer Esiason Foundation, a nonprofit group working to connect leaders in medicine…

Treatment with Orkambi (lumacaftor/ivacaftor) can effectively improve overall health in adolescents and adults with cystic fibrosis, but its use can be risky for those who do not tolerate the therapy, a study of its use in almost 850 CF patients across France found. More than 18% of these patients…

The UNITY prenatal test is highly sensitive and accurate for detecting hereditary diseases such as cystic fibrosis (CF), spinal muscular atrophy (SMA), sickle cell disease, and thalassemias, a new study reports. Developed by BillionToOne, the non-invasive test uses maternal blood to detect CF and…