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…
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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…
People with cystic fibrosis-related diabetes (CFRD) show alterations in the lung microbiome, the community of microbes that live in the lungs, a study from Sweden found. These alterations are characterized by reduced diversity of bacterial types and more imbalanced bacterial composition. The study also demonstrated that patients with CFRD had…
Low- and ultra-low-dose lung CT scans may help monitor how people with cystic fibrosis (CF) respond to CFTR modulator therapies while using less radiation, a study in Italy suggests. After starting Trikafta (ivacaftor, tezacaftor, and elexacaftor), patients showed a nearly 50% reduction in airway thickening and a…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
Among people with cystic fibrosis (CF) who develop kidney failure, getting a kidney transplant may extend survival by nearly two decades compared with other types of care, a new study found. The transplanted kidneys also showed favorable long-term outcomes in people with CF, remaining functional for about 7.2 years…
Both youngsters and teenagers with cystic fibrosis (CF) have abnormal levels of bile acids, the main components of the digestive fluid bile, in their blood compared with healthy children — and these alterations were found to worsen with age — a new study reports. The researchers also identified distinct…
The lungs of infants and children with cystic fibrosis (CF) show an early, overactive immune response driven by neutrophils — a type of immune cell — even in a patient receiving CFTR modulator therapy, a small study suggests. The findings suggest that inflammatory processes that damage the lungs may…
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Recent Posts
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- American Airlines ski event raises more than $1.6M for CF Foundation
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