Gene therapy using circular forms of DNA called plasmids may help treat lung disease in cystic fibrosis (CF), based on early lab studies, regardless of the disease-causing gene mutation, a study suggests. Optimized plasmids boosted production of the fully mature CFTR protein, which is missing or not working properly…
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Trikafta was associated with significant reductions in healthcare use, including hospitalizations related to cystic fibrosis (CF) complications affecting organs and systems outside the lungs, according to an insurance claims analysis. “Results of this study suggest a multisystemic benefit of [Trikafta] therapy with reductions in extrapulmonary…
In people with cystic fibrosis (CF), the timing of organ transplant — lung, liver, or both — relative to infection with the SARS-CoV-2 virus that causes COVID-19 did not affect the risk of severe outcomes, according to a large international study. Patients with poorer lung…
Among people with cystic fibrosis (CF), certain measures of blood vessel health — especially regarding the aorta, the body’s main artery — tend to vary according to biological sex, a new study reports. Researchers think these variations may help explain sex-specific differences in survival outcomes among people with CF,…
Bioversys has entered into an exclusive collaboration and license agreement with Hackensack Meridian Health (HMH) and its Center for Discovery and Innovation to advance a novel class of antibiotics targeting hard-to-treat nontuberculous mycobacteria (NTM) infections, an important concern for people with cystic fibrosis (CF). Under the…
Children with cystic fibrosis (CF) who undergo a liver transplant alone have significantly better survival rates than those who receive a combined liver-lung transplant, according to a new analysis of U.S. transplant data. While both procedures are life-saving options for patients with advanced disease, the study highlights a gap in…
The U.S. Food and Drug Administration (FDA) has expanded the approvals of two Vertex Pharmaceuticals therapies to include additional mutations that cause cystic fibrosis (CF), allowing more people with the condition to become eligible for treatment. The decision concerns Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) and Trikafta (elexacaftor/tezacaftor/ivacaftor). These are CFTR…
Agena Bioscience has launched a new genetic testing panel designed to detect a broader range of mutations that cause cystic fibrosis (CF), the company announced. The CFTR 100+ Panel can analyze more than 100 variants in the CFTR gene, which is altered in CF. It includes all…
American Airlines and its partners have raised more than $1.6 million for the Cystic Fibrosis Foundation (CFF) during the airline’s 41st annual Celebrity Ski event, continuing a four-decade tradition of hitting the slopes to fund a cure. The event, held March 5-8 in Beaver Creek, Colorado, featured…
Kidney stones are commonly detected in children with cystic fibrosis (CF), but are often small and do not require surgical treatment, according to a new study from Turkey. Nearly a fifth of the children evaluated in the study had kidney stones detectable by ultrasound, and even more had evidence…
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