News

Six months of treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor), sold as Trikafta in the U.S., leads to increases in body mass index (BMI) and fat mass in people with cystic fibrosis (CF), a small study has found. However, while BMI increased for both men and women, only women showed a…

Researchers hope a new tool that can provide disease-specific education and resources about a lung transplant for people with cystic fibrosis (CF) will be able to make inroads among patients who’ve historically had poor access to the procedure. The development of the tool, called Take on Transplant, was introduced…

Blocking iron-dependent cell death, a process called ferroptosis, may treat antibiotic-resistant Pseudomonas aeruginosa lung infections in people with cystic fibrosis (CF). “Blocking ferroptosis might be a really promising and unique new way to treat [Pseudomonas aeruginosa] infections,” said Peter A. Jorth, PhD, from Cedars-Sinai Medical Center, California,…

Long-term Trikafta treatment led to sustained improvements in CFTR function and reduced lung symptoms in children, ages 6-11, with cystic fibrosis (CF), according to almost three years of interim data from an open-label extension (OLE) study. There were no signs of lung function decline during the 3.5-year treatment…

Sionna Therapeutics’ second-generation molecules, combined with standard CFTR modulators, fully restored the production and function of the CFTR protein with the most common mutation that causes cystic fibrosis (CF), according to preclinical data. The Series 2 molecules, SION-719 and SION-451, are undergoing investigational new drug (IND) enabling…

Treatment with the inhaled gene therapy 4D-710 led to clinically meaningful quality of life gains for people with cystic fibrosis (CF) in a clinical trial, according to data announced by 4D Molecular Therapeutics (4DMT), the therapy’s developer. Treatment also led to high expression of the CFTR protein…

The first person with cystic fibrosis (CF) has been dosed with CM001, an experimental inhaled therapy being developed by Cystetic Medicines to treat all people with CF, including those who aren’t eligible for modulators. “Dosing the first person with CF represents a critical milestone in our mission to…

One month of treatment with Trikafta, a triple combo of elexacaftor, tezacaftor, and ivacaftor, helped to clear mucus from the airways of adolescents and adults with cystic fibrosis (CF), a study found. Mucus became less thick, which may have helped in preventing its buildup in the airways, increasing…

Six months of treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) significantly improved lung function and nutritional status in two adults with cystic fibrosis (CF) caused by the rare mutation M1101K, according to two cases described in Canada. “Our cases not only demonstrate the therapeutic benefit of [Trikafta] in individuals with non-F508del…

Trikafta, a combination of elexacaftor, tezacaftor, and ivacaftor, lowered the number of Pseudomonas aeruginosa bacteria in the nose and sinuses of people with cystic fibrosis (CF) taking part in a small study. Mucus from the sinuses — air-filled pockets in the skull — can enter the lungs, so…