Nivalis Therapeutics, Inc., a Boulder, Colorado-based clinical stage pharmaceutical company, announced that the first patient has been dosed in the Phase 2 clinical trial of its lead investigational drug, N91115, a stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The purpose of this double-blind, randomized, placebo-controlled, parallel group study…
Protein interactions could be misdirected in people with cystic fibrosis (CF), causing a loss of normal protein function, according to researchers from the Scripps Research Institute (TSRI) in San Diego, California. The new study, titled “∆F508 CFTR interactome remodelling promotes rescue of cystic fibrosis“ appeared online on Nov. 30, 2015, ahead…
The Cystic Fibrosis Foundation’s 2015 Ultimate Golf Experience, which ran four days beginning on Oct. 21, raised $630,000 towards continuing the organization’s mission of promoting disease research and awareness. Over 200 people attended the tournament at the Ritz-Carlton Dove Mountain resort in Tucson, Arizona. The event was hailed as a success and a source of much-needed…
Ever since High Frequency Chest Wall Oscillation (HFCWO) devices were developed to help patients with cystic fibrosis breathe easier, clinical researchers required appropriate parameters to assess the efficacy of HFCWO-based therapies. It is common practice to look at three main indicators of pulmonary and overall health: mucus clearance,…
A new cystic fibrosis study, showing a high-resolution picture of the fungal microbiome of cystic fibrosis patients’ lungs, has identified a genetic basis for pathogen adaptation in a fungus exposed to bacteria. The study, entitled “Global Analysis of the Fungal Microbiome in Cystic Fibrosis Patients Reveals Loss of Function of…
PTC Therapeutics Inc. has announced the completion of patient enrollment in its second Phase 3 clinical trial of the experimental cystic fibrosis therapy Translarnaâ„¢. The clinical trial, named ACT CF, is a placebo controlled study designed to evaluate the efficacy of Translarna (ataluren) in patients, age 6 or older, with nonsense…
Cambridge, Massachusetts based Vertex Pharmaceuticals Incorporated reports that the European Commission (EC) has approved expansion of the indication for its cystic fibrosis drug KALYDECO (ivacaftor), which now includes children aged two to five with cystic fibrosis (CF) and who have one of nine gating mutations in the cystic…
Concert Pharmaceuticals, Inc., a clinical stage pharmaceutical company that focuses on the development of new small molecule drugs through its DCE (deuterated chemical entity) Platform®, has announced the beginning of Phase 1 multiple ascending dose clinical trial with its cystic fibrosis treatment CTP-656 (deuterium-modified ivacaftor). The announcement follows recent news…
Encouraging results of a study presented by the Katholieke Universiteit (KU) Leuven Laboratory for Molecular Virology and Gene Therapy at Leuven, Belgium indicate that improved gene therapy treatment can cure mice with cystic fibrosis (CF). The researchers note that cell cultures from CF patients also respond well to the…
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for AB569 from Arch Biopartners Inc. as a treatment for Pseudomonas aeruginosa (P. aeruginosa) pulmonary infections in patients with cystic fibrosis. The Orphan Drug Designation is a crucial step for the development of new medicines for…
Visit the Cystic Fibrosis News Today forums to connect with others in the CF community.
Get regular updates to your inbox.