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Six months of treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) led to significant improvements in lung function and a self-reported easing of respiratory symptoms for children with cystic fibrosis (CF) ages 6 to 11 in an observational study of its real-world use. Felix Ratjen, MD, PhD, division chief of pediatric respiratory…

A single dose of 4D-710, an aerosol gene therapy candidate developed by 4D Molecular Therapeutics (4DMT) for adults with cystic fibrosis (CF), was reported to be safe and restored CFTR protein production in lung tissue samples from three patients enrolled in a Phase 1/2 clinical trial. Interim trial…

Treatment with Trikafta leads to an increase in body weight and cholesterol levels among people with cystic fibrosis (CF), according to a new study into the impacts of the approved CF triple therapy. The study found gains in body mass index — BMI, a measure of body weight…

With continual advances in treatment, more people with cystic fibrosis (CF) are surviving well into adulthood and by 2040, nearly three-quarters of these patients in the U.S. will be adults. That’s according to a study, “Cystic Fibrosis in the United States by 2040: a population landscape…

Women and girls with cystic fibrosis (CF) who are being treated with antibiotics due to a pulmonary exacerbation tend to have better lung function compared with male patients, but report more severe symptoms, new data show. Kristina Montemayor, MD, a professor at Johns Hopkins University, shared the findings at…

CFTR modulators, a highly effective therapy for cystic fibrosis (CF), do not appear to affect fertility outcomes for men using them, data from surveys of these patients indicate. Miscarriages during pregnancy for a woman whose partner is using modulator therapy and congenital anomalies for the child were not higher than expected, researchers reported.

Note: This story was updated Nov. 9 to note that experiments done with Sionna’s CFTR modulators used a different investigational NBD1 stabilizer than SION-638. SION-638, a first-in-class small molecule that is being developed by Sionna Therapeutics, improved the stability and functionality of the CFTR protein  in the most common…

Exposure to tobacco smoke may reduce the effectiveness of Kalydeco (ivacaftor), a CFTR modulator that is widely approved to treat cystic fibrosis (CF) caused by specific mutations. That’s according to data shared by Gabriela Oates, PhD, at the 2022 North American Cystic Fibrosis Conference, taking place Nov. 3–5…

A novel scoring system may be used to classify the severity of lung disease among children with cystic fibrosis (CF) in their first years of life. Leslie Huang, a medical student at the University of Wisconsin, discussed the system at the 2022 North American Cystic Fibrosis Conference in Philadelphia,…

Treatment with Trikafta continues to be safe and well-tolerated, benefiting people with cystic fibrosis (CF) ages 12 and up who have been on treatment for almost three years. But benefits may also extend to children as young as 2. That’s according to data that will be shared at…