PTC Therapeutics, Inc. recently announced the recipients of its global STRIVE Awards (Strategies to Realize Innovation, Vision and Empowerment) program, which supports nonprofit associations assisting the cystic fibrosis (CF) community. The STRIVE Program recognizes the vital role patient advocacy groups play in giving voice to individuals affected by these rare…
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ProMetic Life Sciences Inc. recently announced that Health Canada has approved the initiation of a clinical trial assessing the company’s anti-fibrotic lead drug candidate, PBI-4050, in patients with cystic fibrosis (CF). The Phase 2 study, which is expected to begin in shortly, aims to evaluate the drug’s effects on pancreatic and lung function…
Nivalis Therapeutics announced the dosing of a first patient in a Phase 2 clinical trial evaluating the efficacy and safety of its investigational drug N91115, in combination with ivacaftor (Kalydeco), in people with cystic fibrosis (CF). CF is a life-shortening genetic disease, characterized by mutations in the CFTR gene that lead to defective chloride…
Silencing a gene involved in the processing of the mutant CFTR protein in cystic fibrosis (CF) improved folding and increased the amount of functional protein that reached the plasma membrane — reaching clinically relevant levels when used in combination with the modulator drug VX-809 (lumacaftor). The results, from experiments in…
Researchers at the Queen’s University Belfast developed a new compound that may change how lung ailments in cystic fibrosis (CF) are treated. The potential medicine was shown in an early study to improve airway hydration and increase mucus clearance, results that suggest a mechanism to potentially delay or prevent lung disease independently of the…
AbbVie welcomes undergraduate and graduate students with cystic fibrosis (CF) to apply for the 2016 AbbVie CF Scholarship, awarding $3,000 for education-related expenses. Applications will be accepted until May 27 and are available online at www.AbbVieCFScholarship.com. The CF community in the U.S. includes approximately 30,000 children and adults, and about half…
Galapagos NV recently announced the start of a Phase 1 clinical trial to address the safety and tolerability of its potentiator candidate, GLPG2451, to treat cystic fibrosis (CF). CF is caused by mutations in the CFTR gene that lead to an abnormal transport of chloride across cell membranes, resulting in defective hydration of…
International Biophysics Corporation recently announced the publication of new data regarding the effectiveness of its AffloVest airway clearance technology in patients with cystic fibrosis. The paper, “Lung function improvement with AffloVest® HFCWO use: a clinician’s perspective on PFT score data from 12 patients with cystic fibrosis,” was published on the…
People whose cystic fibrosis was detected through screening programs for newborns were found to have better growth throughout puberty and superior adult height, compared to those whose CF was diagnosed after they began exhibiting symptoms, researchers at the University of Wisconsin–Madison reported. The study, based on analyzed data from the Wisconsin Randomized Clinical Trial, “Pubertal Height…
Researchers at the University of Pittsburgh Center for Vaccine Research (CVR) have developed an engineered antimicrobial peptide effective against co-infection by bacterial microfilm and a virus, and as such is a possible new therapeutic agent against chronic and antibiotic-resistant infections. Importantly, the potential drug was tested against Pseudomonas aeruginosa, one of the primary and more dangerous…
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