In the cystic fibrosis (CF) community, May is an important month — one set aside by the Cystic Fibrosis Foundation to educate and raise awareness across the U.S. of the disease and its impact on people’s lives as part of National CF Awareness Month. Among these efforts is a highly visible window display…
News
The Cystic Fibrosis Foundation, a leading supporter of research funding for new cystic fibrosis (CF) treatments with the goal of finding a cure, announced the appointment of Marc Ginsky as its new executive vice president and chief operating officer. Ginsky will report to the organization’s president and chief executive officer, Dr.
Galapagos NV and AbbVie recently expanded their collaborative agreement in cystic fibrosis (CF) to mirror the successful growth of their CF portfolio, with an ultimate goal of developing a triple combination therapy able to treat 90 percent of all cystic fibrosis patients. Triple combinations of CF compounds already in the companies’…
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…
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…
European Medicines Panel Favors ‘Orphan Product’ Designation for Drug to Treat CF Lung Infections
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…
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