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…
Viral respiratory tract infections in young children with cystic fibrosis tend to be mild and not require hospitalization, a study found. Infections caused by respiratory syncytial virus (RSV) were rare and did not lead to complications, suggesting that there is no need for preventive medication such as…
The U.S. Food and Drug Administration (FDA) has designated Vast Therapeutics’ BIOC11 a Qualified Infectious Disease Product (QIDP) for the treatment of people with cystic fibrosis (CF) who have chronic pulmonary infections due to Pseudomonas aeruginosa. The QIDP designation makes BIOC11 eligible for priority-review and fast-track statuses…
Scientists have discovered a new way to treat cystic fibrosis (CF) that involves delivering artificial proteins to patients’ lung cells to replace the faulty cystic fibrosis transmembrane conductance regulator (CFTR) protein. The finding was reported in the study, “Anion carriers as potential treatments for cystic fibrosis: transport in…
The Western Hemisphere’s largest gathering of experts in cystic fibrosis gets underway later this month in Nashville, Tennessee — site of the 33rd annual North American Cystic Fibrosis Conference (NACFC). The Oct. 31–Nov. 2 event, sponsored by the Cystic Fibrosis Foundation (CFF), will attract more than 5,000 delegates…
Vertex Pharmaceuticals has entered a strategic collaboration with Ribometrix to discover potential RNA-targeted therapies for serious diseases. Vertex is a leader in the market of CFTR modulators for cystic fibrosis (CF), and is the developer of Kalydeco (ivacaftor), Orkambi (ivacaftor/lumacaftor), and Symdeko (tezacaftor/ivacaftor combination therapy), three approved CF…
Using a simple shirt — a smart one — is closer to becoming a common method to measure lung function in patients with respiratory conditions, including chronic obstructive pulmonary disease (COPD) and cystic fibrosis. A new study conducted by researchers from Radboud University Medical Center and University of Twente,…
Inhibition of the interleukin-1 receptor — to block thick mucus production and inflammation — is a potential strategy to treat cystic fibrosis (CF) in the early stages of the disease before harmful chronic bacterial infections, a study suggests. The study, “…
Voting for two students to receive two scholarships — valued at $22,000 each — is now open to the public until Oct. 9, pharmaceutical company AbbVie has announced. After the voting period, AbbVie’s 2019 Thriving Undergraduate Scholarship will be awarded to an undergraduate student with CF, and its Thriving…
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