Aisha I Abdullah PhD,  —

Children and adolescents on Symdeko (tezacaftor/ivacaftor) and Orkambi (ivacaftor/lumacaftor) for cystic fibrosis (CF) had higher treatment adherence rates compared with adults given the same CFTR modulators, a nationwide study in the U.S. found. The study also showed that adherence — patients choosing to follow through with a prescribed treatment —…

Patients in the U.S. with cystic fibrosis (CF) and advanced lung disease are less likely to receive a lung transplant and twice as likely to die before having transplant surgery than their counterparts in Canada, a new study finds. Moreover, the difference was more pronounced…

A review of 57 real-world studies found that people with cystic fibrosis (CF) who take Kalydeco (ivacaftor) experience improvements in lung function, nutrition, and quality of life (QoL). Reduced healthcare system use, lower Pseudomonas…

A clinical study found that in youth with cystic fibrosis (CF) who are in good physical condition, resistance training favorably regulates heart rate variability (HRV), an indicator of the ability to adjust to stress. HRV, a measure of the variation in…

Krystal Biotech is expecting to start a Phase 1 clinical trial by early fall to test KB407 for cystic fibrosis (CF), after the investigational inhaled gene therapy was found safe and well-tolerated in nonhuman primates. According to Krystal, the positive…

Treatment with MS1819 for exocrine pancreatic insufficiency (EPI) — when the pancreas does not release enough digestive enzymes and the body doesn’t receive proper nutrition — was found to be safe and well-tolerated among cystic fibrosis (CF) patients in the OPTION 2 trial, top-line results show. However, MS1819 was…

Patient enrollment has been completed for a Phase 2 clinical trial testing the investigational therapy MS1819 in combination with pancreatic enzyme replacement therapy, called PERT, for severe exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). Pancreatic insufficiency, known as EPI, is a complication of CF characterized by…

Blood C-reactive protein (CRP) and calprotectin levels can reliably identify pulmonary exacerbations in cystic fibrosis (CF) patients, making them promising diagnostic biomarkers, a pilot study has found. Although further investigations are necessary to determine their clinical applicability, the researchers note that “a step-wise algorithm that…

The U.S. Food and Drug Administration (FDA) has accepted for review an application to expand the use of Trikafta as a treatment in children with cystic fibrosis (CF) ages 6 to 11 with at least one F508del mutation in the CFTR gene (the gene defective in…

People with cystic fibrosis (CF) and their families are largely satisfied with the convenience and quality of care received using telehealth models initiated during the COVID-19 pandemic, a survey study from Australia finds. The study, “…