CYSTIC FIBROSIS

This study tested a drug called miglustat in 16 adults with cystic fibrosis who have a specific genetic mutation (F508del). The goal was to see if miglustat could help restore the function of a faulty protein that controls salt and water movement in the lungs. Researchers measure…

This study followed 852 people with cystic fibrosis (age 12 and older) who were taking the drug combination ivacaftor+lumacaftor (Orkambi) for one year. Researchers wanted to see how often patients stopped treatment, why, and how the drug affected lung function and nutrition. The…

This study tested a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) in 70 toddlers with cystic fibrosis who are 12 to 24 months old. The goal was to see how the drug moves through the body, whether it is safe, and if it helps reduce sweat chloride levels, a key measure…

This study looked at the long-term safety of a triple combination therapy (VX-445, tezacaftor, and ivacaftor) for people with cystic fibrosis who have the F508del mutation. 507 participants who completed earlier studies were enrolled. The main goal was to check for side effects a…

This study tested a single dose of a virus mixture (bacteriophages) given intravenously to adults with cystic fibrosis who have a chronic lung infection with Pseudomonas aeruginosa. The goal was to see if the treatment is safe and can reduce the amount of bacteria in the lungs. T…

This study tested whether a noninvasive ultrasound device called FibroScan can accurately measure liver scarring (fibrosis) in children. Currently, the only reliable method is a liver biopsy, which involves inserting a needle into the liver. The study included 264 children aged 6…

This study measured how CFTR modulator treatment (Kaftrio) changes nose and sinus symptoms and quality of life in 27 children aged 6 to 11 with cystic fibrosis. Researchers used a special questionnaire (SN-5) before and one year after starting treatment. The goal was to see if th…

This study looked at blood neutrophils (a type of immune cell) in 47 adults with cystic fibrosis to see how chronic infection and certain treatments affect these cells. Researchers compared cells from patients with those from healthy blood donors. The goal was to learn more about…

This study followed 79 infants with cystic fibrosis under 6 months old to see how their pancreas function changed during the first year of life. Researchers measured a stool enzyme called fecal elastase-1, which indicates how well the pancreas is working. The goal was to understa…

This study looked at whether a test using cells from the nose can predict how well the drug Orkambi works for people with cystic fibrosis. Researchers collected nasal cells from 91 participants before treatment and measured how the drug improved cell function. They then compared …

This study looked at sputum (mucus coughed up from the lungs) from 83 adults with cystic fibrosis who were hospitalized for a sudden worsening of their lung condition. The goal was to find genetic markers of antibiotic-resistant bacteria and see how they relate to treatment outco…

This study looked at whether adding an inhaled sugar solution (Bronchitol) helps clear mucus from the lungs of people with cystic fibrosis who already take a powerful medication (E/T/I). Fourteen adults with moderate to severe lung disease participated. The goal was to see if the…

This study looked at whether people with cystic fibrosis (CF) have natural protection against COVID-19. Researchers tested blood samples from 75 children and adults with CF in Europe for SARS-CoV-2 antibodies over two years. They compared antibody levels after natural infection v…

This study tested whether a voice journaling app called Kintsugi is easy and acceptable for teens aged 14-18 with cystic fibrosis to use. Twenty participants used the app for three months, and researchers tracked how often they journaled and their satisfaction. The goal was to ga…