Concert Pharmaceuticals, Inc., which is developing new small molecule drugs through its DCE (deuterated chemical entity) Platform, recently announced positive results from a Phase 1 clinical trial of its cystic fibrosis (CF) treatment, CTP-656 (deuterium-modified ivacaftor). The data, which included a positive safety and tolerability profile, support the continuing development of CTP-656 as…
News
Pulmonary exacerbations have a negative impact on the general health-related quality of life in people with cystic fibrosis (CF), a study reported, stressing the importance of preventing such flares in this group. Assessing general, as opposed to disease-specific, health-related quality of life measures are needed when comparing different disease conditions and determining the usefulness of…
ACT.md recently reported positive preliminary results from a multiyear project — in partnership with Boston Medical Center and Baystate Health — using the company’s “Collaborative Consultative Care Coordination Program” (4C) to measure improvements in quality of life, family engagement, and healthcare costs for children affected by medically complex conditions like cystic fibrosis. First results…
The European Medicines Authority’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending the designation of orphan medicinal product to Arch Biopartners Inc.‘s AB569 as a potential treatment of infections in cystic fibrosis (CF) patients. AB569 was invented at the University of Cincinnati (UC) in the laboratory…
A scientific paper published by Spanish researchers discusses a strategy for treating the difficult-to-target F508del-CFTR mutation, a cause of cystic fibrosis (CF). The report, “Manipulating proteostasis to repair the F508del-CFTR defect in cystic fibrosis,“ appeared on March 14 in the journal Molecular and Cellular Pediatrics. CF…
According to the study “The Role of Computed Tomography in Monitoring Patients with Cystic Fibrosis” published in the Polish Journal of Radiology, computed tomography (CT), an imaging technique that is commonly used for the evaluation of cystic fibrosis (CF) in individual patients, can play a critical…
Two Texas surgical teams recently performed an extremely rare, lungs-and-liver double organ transplant at the University of Texas (UT) Southwestern Medical Center’s William P. Clements Jr. University Hospital. The operation, the first successfully completed in North Texas and only the 75th such surgery in the United States, gave 25-year-old Josiah…
A Chicago Medical School review explored the scientific evidence behind three natural compounds — genistein, curcumin, and resveratrol — often proposed for the treatment of cystic fibrosis (CF). The review concluded that while the mechanisms by which these substances function should be further investigated as potential drug treatments, there is little evidence…
The Cystic Fibrosis Foundation announced that it welcomes the recent creation of a Cystic Fibrosis Caucus in the U.S. Senate, several years after a similar caucus was established in the lower branch of the U.S. Congress, the House of Representatives. The foundation also thanked the bipartisan work of two…
Pulmatrix, Inc.’s chief scientific officer, Dr. David L. Hava, described in an article recent advances in drug delivery strategies — such as nebulizers for inhaled delivery — that are helping to treat bacterial and fungal lung infections, and are particularly relevant to people with diseases like cystic fibrosis (CF). The article, “…
Your CF Community
Visit the Cystic Fibrosis News Today forums to connect with others in the CF community.