Enterprise Therapeutics recently announced that it has closed a £4 million (about $5 million) financing deal to accelerate the development of three new treatments — all muco-regulatory therapies — for cystic fibrosis (CF), particularly, but also for chronic obstructive pulmonary disease (COPD) and severe asthma. “Respiratory disease remains an area of high unmet medical…
News
Celtaxsys recently announced that a second study describing the results of Phase 1 clinical trials evaluating acebilustat, an oral treatment for cystic fibrosis (CF), has been published. A Phase 2 study is underway and currently enrolling patients in the U.S., Canada and Europe. The research article is titled “Phase 1…
Children with cystic fibrosis (CF) who carry the F508del mutation in both CFTR gene copies showed improved lung function after treatment with Orkambi (ivacaftor/lumacaftor), according to results in a Phase 3 study developed by Vertex Pharmaceuticals. The study, in pediatric patients ages 6–11, evaluated the effect of Orkambi…
Scientists at Pennsylvania State University have developed a relatively inexpensive, easy-to-use system for diagnosing cystic fibrosis (CF), a technique that uses florescent light to measure levels of chloride in perspiration, which are known to be elevated in CF patients. The research was supported by the National Institute of Biomedical Imaging and…
ProQR Therapeutics is reporting that an early clinical study of QR-010 in cystic fibrosis patients homozygous for the ΔF508 mutation in the CFTR gene met its primary endpoint. After four weeks of treatment, these patients showed a positive change in total chloride response, meaning that the CFTR protein was again working. In patients heterozygous for…
Verona Pharma recently announced that it has received its second Venture and Innovation Award from the U.K. Cystic Fibrosis Trust. The award will help fund a Phase 2 clinical trial to investigate RPL554 as a possible treatment of cystic fibrosis (CF). The trial, which expects to start recruiting CF patients during the first…
Early lab and animal studies suggest that Spyryx Biosciences’ drug candidate SPX-101 will be a safe and effective way of improving mucus clearance in patients with cystic fibrosis (CF), regardless of the underlying mutation(s) causing their disease. The data were presented at the 30th Annual North American Cystic Fibrosis…
The Boomer Esiason Foundation (BEF) announced the Sacks for CF Scholarship for 2016-17, which are funding grants for college students who have cystic fibrosis (CF). Awards for undergraduates and graduates of $3,000 to $10,000 are given annually to 30 college students who strive for adherence to daily CF therapy,…
Alcresta Therapeutics announced that its product RELiZORB effectively increased fat absorption in cystic fibrosis (CF) patients receiving enteral tube feeding and who have trouble breaking down and absorbing these molecules, resulting in reduced frequency and severity of gastrointestinal symptoms among those patients. The findings were presented at the 30th…
A transition from youth to adult cystic fibrosis (CF) care needs to be guided by formal programs to maximize patient satisfaction and health outcomes. For successful transitions, skills essential to managing independent adult care also need to be developed, and assessing such skills before attempting a transfer would be wise. The “…
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