Iqra Mumal MSc,  —

Patients with cystic fibrosis (CF) in the Republic of Ireland will be among the first in Europe to benefit from Vertex Pharmaceuticals’ triple combination regimen composed of elexacaftor (VX-445), tezacaftor, and Kalydeco (ivacaftor), upon its approval by the European Medicines Agency. Following Vertex’s negotiations with the Republic of…

High levels of the peptide copeptin are associated with increased severity of cystic fibrosis (CF) in children, a study shows. These results suggest that copeptin can be used as a biomarker to identify patients who could benefit from more aggressive treatment. The study, titled “Evaluation of…

Researchers in Israel and the U.S. have developed a new technology that can be used to unclog and eliminate mucus secretions from the airways of patients with various respiratory diseases, including cystic fibrosis (CF). The announcement was made by BGN Technologies, the technology company of Ben-Gurion University…

The Comité Économique des Produits de Santé (CEPS) in France has approved national reimbursement of Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) for patients with cystic fibrosis (CF) who are 2 years of age or older, with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)…

The National Institutes of Health (NIH) has granted $2.7 million to Michigan State University (MSU) scientist Robert Quinn to investigate, in collaboration with Spectrum Health, the role of anaerobic bacteria in the development of flare-ups among patients with cystic fibrosis (CF). Anaerobic bacteria are a type…

Proteostasis Therapeutics (PTI) has completed patient enrollment in a Phase 2 clinical trial evaluating its cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies in cystic fibrosis (CF) patients who have at least one copy of the F508del mutation in the CFTR gene (the defective gene in CF…

Arrevus received a Small Business Research Loan from the North Carolina Biotechnology Center to support the development of the company’s Phase 3 therapeutic candidate ARV-1801 for the treatment of pulmonary exacerbations in patients with cystic fibrosis (CF). The Small Business Research Loan program grants investments of $75,000—$250,000…

Vertex Pharmaceuticals has reached an agreement with England’s National Health Service (NHS), allowing patients there access to all Vertex cystic fibrosis (CF) treatments currently approved in Europe at low or no out-of-pocket cost. The agreement between the NHS and Vertex appears to end a long-standing dispute over the company’s pricing…

Ultrasound imaging can be a reliable diagnostic tool for early detection of liver disease in children with cystic fibrosis (CF), a study finds. Using this strategy can help clinicians identify CF patients most at risk for liver damage caused by cirrhosis (tissue scarring), researchers suggest. These findings were reported in…

The Cystic Fibrosis Foundation (CFF) will fund the preclinical development of two gene therapy candidates, Talee Bio’s TL-101 and TL-102, for the treatment of cystic fibrosis (CF), the company announced. Talee will receive up to $4.5 million from CFF to help accelerate the development of both these gene therapies…