Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…
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A young girl with cystic fibrosis (CF) and acute recurrent pancreatitis was able to eat some food normally and experienced relief of her abdominal symptoms after undergoing a complete removal of her pancreas and a transplant of pancreatic cells into her liver, according to a case report. At…
Pulmonary infection with a specific species of the Mycobacterium avium complex (MAC), called Mycobacterium intracellulare, can be aggressive in people with cystic fibrosis (CF), with a greater loss of lung function evident but a lesser incidence of a secondary lung infection, a single-site study reports. This species also responded best…
The U.S. Food and Drug Administration (FDA) has granted priority review to Vertex’s new drug application for its triple combination — elexacaftor (VX-445) plus tezacaftor and Kalydeco (ivacaftor) — for cystic fibrosis (CF) patients who cannot use the company’s other disease-modifying treatments or don’t benefit as intended.
Path BioAnalytics (PBA), a biotechnology company based in North Carolina, has licensed the rights to further develop cavosonstat (N91115), a targeted therapy for cystic fibrosis (CF), from the Chinese company Laurel Therapeutics. Cavosonstat is a small molecule designed to increase the stability of the CFTR protein, defects of which cause CF.
A defect in cellular metabolism is the root cause for the high incidence of lung infections caused by the bacteria Pseudomonas aeruginosa in patients with cystic fibrosis (CF), a study has found. Results from the study, “CFTR-PTEN–dependent mitochondrial metabolic dysfunction promotes Pseudomonas aeruginosa airway infection,” were published in…
Trained sniffer dogs are able to detect Pseudomonas aeruginosa, the most common bacteria found in people with cystic fibrosis (CF), in liquid cultures even at very low concentrations, a study shows. This potentially could be useful for detecting infections earlier in CF patients — and without the need…
Molecules used by a bacterial species against other microbes, called rhamnolipids, can bolster the ability of a standard antibiotic to penetrate treatment-resistant strains of Staphylococcus aureus by essentially making holes in the membrane of its cells, a new study confirms. The study, “…
Cystic fibrosis (CF) patients treated with Kalydeco (ivacaftor) who have the G551D mutation reported better health-related quality of life (HRQoL) and symptom relief than those on standard treatment who carry the F508del mutation in the CFTR gene, according to a real-world study. The research, “Patient-reported…
High blood levels of the enzyme alanine aminotransferase (ALT) — a biomarker of liver damage — might indicate a higher risk of rising blood sugar levels and cystic fibrosis-related diabetes (CFRD), especially in men, a study found. These data suggest that ALT could serve as a marker of CFRD risk…
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