The Cystic Fibrosis Foundation is making a new investment of up to $11 million in 4D Molecular Therapeutics (4DMT) to support the development of 4D-710, the company’s inhaled gene therapy for cystic fibrosis (CF) that is currently in clinical development. The funding, which builds on earlier support,…
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While treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) helps people with cystic fibrosis (CF) gain weight, a tendency toward unhealthy diets may pose new risks for patients — warranting “close monitoring and interventions to prevent excessive weight gain” — a study from Norway reports. People with CF in the European nation who…
As a child, Ella Balasa was no stranger to hospital stays. Diagnosed with cystic fibrosis (CF) at 18 months, she faced persistent lung infections that often required weeks-long courses of antibiotics, multiple times a year. “I was always in the hospital needing treatment,” Balasa, now 33 and living in…
Treatment with the antibiotic azithromycin improves breathing while slowing lung function decline over time in children and teens with cystic fibrosis (CF), according to a meta-analysis of published studies. “These findings not only provide a new perspective for clinical treatment but also reveal potential mechanisms of azithromycin in the…
The experimental anti-inflammatory therapy brensocatib was generally well tolerated in adults with cystic fibrosis (CF), according to data from a small clinical trial aimed at aiding the design of future studies of the therapy. Brensocatib’s pharmacological profile in CF patients is similar to its profile in people without CF,…
The Cystic Fibrosis Foundation has made an additional $3 million investment in Recode Therapeutics to support clinical development of RCT2100, an investigational therapy for cystic fibrosis (CF) that is currently being tested in patients who cannot take CFTR modulators. The CF Foundation had previously invested $15 million…
One-year treatment with Trikafta — a widely used, approved medication for people with cystic fibrosis (CF) — significantly eases symptoms and improves outcomes, regardless of whether or not patients were previously treated with other CFTR modulators, a new real-world study reports. Among other benefits, Trikafta — a combination…
People living with cystic fibrosis (CF) find it easier to maintain good nutrition when they have access to personalized care from dietitians and nutritionists, an interview-based study found. But food costs, health problems, and lack of support from care teams can be barriers to achieving nutrition goals, the researchers said.
The Unity Fetal Risk Screen, a test that uses a pregnant person’s blood to evaluate the fetus’s risk of cystic fibrosis (CF), accurately detected all cases of CF in a study involving more than 100,000 pregnant people. The study found that most CF cases identified by the test are…
A trio of scientists is being honored for research contributions that helped lead to the development of Trikafta, a triple-drug combination medication for people with cystic fibrosis (CF). The three winners of this year’s Lasker~DeBakey Clinical Medical Research Award are Paul Negulescu, PhD, senior vice president at Vertex…
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