Tag: cystic fibrosis

1. In this multicenter, phase 3 study of cystic fibrosis patients who were previously unresponsive to modulator regimens, triple-combination therapy with the next-generation corrector elexacaftor resulted in a significantly improved percentage of predicted forced expiratory volume in one second versus placebo after 4 and 24 weeks. 2. Improvements were also...
1. A G551D missense mutation in the cystic fibrosis transmembrane conductance receptor (CFTR) is a genetic cause of cystic fibrosis (CF). 2. In utero administration of the CFTR-potentiating drug, VX-770 (ivacaftor) in a ferret genetic model of cystic fibrosis provided protection from CF pathologies in the pancreas, intestine, and male...
Effect of Oral Capsule – vs Colonoscopy-Delivered Fecal Microbiota Transplantation on Recurrent Clostridium difficile Infection The most efficacious means of reducing recurrent clostridium difficile infection (RCDI) is by way of fecal microbiota transplantation (FMT). It is unclear, however, whether clinical efficacy varies with route of delivery, either by colonoscopy or oral capsule....
1. Absolute change in predicted forced expiratory volume in 1 second (FEV1) improved significantly over a 4 and 8-week period among patients assigned tezacaftor-ivacaftor therapy or ivacaftor compared to placebo. 2. Those randomized to the tezacaftor-ivacaftor or ivacaftor treatment groups exhibited significant improvement in cystic fibrosis questionnaire-revised (CFQ-R) respiratory domain...
1. Thymosin α1 (Tα1), an immunomodulatory drug currently in use, decreased lung inflammation in a mouse model of cystic fibrosis (CF). 2. In mice, administration of Tα1 improved the localization and function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Evidence Rating Level: 1 (Excellent) Study Rundown: CF is a disorder...
1. After adjusting for risk factors associated with survival, Canadian patients had a median age of survival that was 10 years greater than in the United States. 2. The authors hypothesized that differential access to transplantation, increased post-transplant survival, and differences in health care systems may partially explain the Canadian...
1. In patients with cystic fibrosis, monthly use of non-viral pGM169/GL67A gene therapy was associated with a significant, but modest, benefit in forced expiratory volume in one second (FEV1) at one year, compared to a decline with placebo. 2. Though this treatment effect was significant, it represented stabilization in...
1. Mucus from cystic fibrosis patients had a rope-like structure and high elasticity due to chemical cross-links between mucin proteins. 2. A compound designed to break down mucin cross-links was more effective in vitro than a currently available drug and was safe in mice. Evidence Rating Level: 2 (Good) Study Rundown: In...

Patient Basics: Cystic Fibrosis

Originally published by Harvard Health. What Is It? Cystic fibrosis is an inherited disease. It causes cells to produce mucus that is sticky and thicker than normal. This mucus builds up, particularly in the lungs and organs of the digestive tract. Cystic fibrosis affects many parts of the body, including the lungs,...
1. From 2000 to 2010, adjusted mortality for cystic fibrosis (CF) patients dropped by 17% (1.8% per year). 2. Median survival for those born with CF in 2010 could exceed 50 years if mortality continues to decrease at a similar pace. Evidence Rating Level: 2 (Good) Study Rundown: Cystic fibrosis (CF) is...