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…
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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…
The Scottish Medicines Consortium (SMC) is recommending against bringing two of Vertex Pharmaceuticals’ approved cystic fibrosis (CF) treatments — Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) — into routine use by adding them to the country’s national health system. Each treatment’s “cost in relation to its health…
Lung transplants are the ultimate therapeutic strategy for many people with cystic fibrosis (CF) and other chronic lung diseases, but they are scarce due to the severe shortage of donor organs, which currently is the only way of obtaining a lung. Research in the lab of Laura Niklason, MD,…
A minimally invasive imaging device that generates high-resolution images of nasal airways, including the cilia and mucus, allows researchers to visualize changes at the cellular level that underlie cystic fibrosis (CF) in patients. The data confirms that mucus dehydration and abnormalities of the airway’s epithelia, as well as…
A 10th anniversary screening of the documentary “65_RedRoses” will take place on Sunday, Sept. 8, at the Vancouver Playhouse Theatre to raise funds for the Transplant Research Foundation of British Columbia (TRFBC). “65_RedRoses” is an award-winning documentary that was directed by Nimisha Mukerji and Philip Lyall. It follows Eva…
The Cystic Fibrosis Foundation (CFF) will give up to $15 million to Arcturus Therapeutics to advance the development of an experimental RNA therapy, called LUNAR-CF, to treat CF patients with all types of mutations in the CFTR gene. The funding will establish a multi-year program aimed at boosting the development…
A single dose of MRT5005, Translate Bio‘s first-in-class messenger RNA (mRNA) therapy for the treatment of cystic fibrosis (CF), improves patients’ lung function, interim data from a Phase 1/2 trial shows. While stressing these are early results, the researchers called the findings “promising.” CF is a…
Treatment for three years with Kalydeco (ivacaftor) reduces the risk of lung infection by serious pathogens — including Pseudomonas aeruginosa and Staphylococcus aureus — in people with cystic fibrosis (CF), a U.K. study reports. The study, “Ivacaftor Is Associated with Reduced Lung Infection by Key Cystic Fibrosis…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
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