Topic cystic fibrosis

Intestinal obstructions have many causes, some of which are genetic. In extreme cases, such as in some instances of cystic fibrosis (CF), intestinal blockages are found even at birth. Ben-Gurion University of the Negev and Soroka University Medical Center Prof. Ohad Birk’s research group has discove more

By developing software that uses 3-D models of proteins involved in cystic fibrosis, a team of scientists at Duke University has identified several new molecules that may ease the symptoms of the disease.  Computer algorithms created by the team predict how well a given molecular structure will bloc more

Horizon Discovery announced its collaboration with the Rosalind Franklin University of Medical Sciences (RFUMS) to develop a human cellular disease model of cystic fibrosis. The agreement focuses on the use of Horizon’s GENESIS™ precision genome editing technology to reconstitute a specific mutation more

Up until the new millennium, the pharmaceutical industry grew stronger by 11%. However, the blockbuster model is increasingly at risk and the declining growth rates in the industry require a search for new strategies. In this context, orphan drugs are becoming increasingly important more

Pseudomonas elastase (LasB) is a key drug target in various chronic and intractable infections. A library of 160 potential, in-house synthesised, elastase inhibitors were screened using a fluorescence assay with the internally quenched substrate Abz-Ala-Gly-Leu-Ala-Nba. Enzymatic kinetic measurements were done on a FLUOstar OPTIMA and Km and Vmax values were calculated for each inhibitor using the MARS Data Analysis software more

A significant proportion of disease-causing mutations affect precursor-mRNA splicing, inducing skipping of the exon from the mature transcript. Using F9 exon 5, CFTR exon 12 and SMN2 exon 7 models, we characterized natural mutations associated to exon skipping in Haemophilia B, cystic fibrosis and more

Clin Microbiol Infect Abstract Despite intensive eradication therapy, some CF patients with early Pseudomonas aeruginosa infection rapidly develop a chronic infection. To elucidate factors associated with this persistence, bacterial characteristics of early P. aeruginosa isolates were analy more

Abstract Cystic fibrosis transmembrane conductance regulator (CFTR) mutation is associated with a phenotypic spectrum that includes cystic fibrosis (CF). The disease liability of some common CFTR mutations is known, but rare mutations are seen in too few patients to categorize unequivocally, maki more

Introduction Global Markets Direct’s, 'Cystic Fibrosis - Pipeline Review, H1 2012', provides an overview of the Cystic Fibrosis therapeutic pipeline. This report provides information on the therapeutic development for Cystic Fibrosis, complete with latest updates, and special features on late-stag more

Introduction Global Markets Direct’s, 'Cystic Fibrosis - Pipeline Review, Q1 2011', provides an overview of the Cystic Fibrosis therapeutic pipeline. This report provides information on the therapeutic development for Cystic Fibrosis, complete with latest updates, and special features on late-stage more

Galapagos NV (Galapagos) is a publicly held research focused biopharmaceutical company. It is engaged in discovery and development of disease modifying drugs. The company develops GLPG0259 that targets protein kinase MAPKAPK5, as its leading drug candidate for the under initial stages of clinical tr more