Vertex's cystic fibrosis therapy meets goal of late-stage studies

Vertex Pharmaceuticals Inc said on Wednesday its triple-combination therapy showed improvement in lung function for people with cystic fibrosis (CF) in two late-stage studies.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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The devastating inherited disease cystic fibrosis (CF) is caused by mutations of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) anion channel. The recent approval of the CFTR potentiator drug ivacaftor (Vx-770) for the treatment of CF patients has marked the advent of causative CF therapy. Currently, thousands of patients are being treated with the drug, and its molecular mechanism of action is under intensive investigation. Here we determine the solubility profile and true stimulatory potency of Vx-770 towards wild-type (WT) and mutant human CFTR channels in cell-free patches of membrane. We find that its ...
Source: eLife - Category: Biomedical Science Tags: Structural Biology and Molecular Biophysics Source Type: research
by David Hasler, Pinelopi Anagnostopoulou, Sylvia Nyilas, Philipp Latzin, Johannes Schittny, Dominik Obrist The multiple-breath washout (MBW) is a lung function test that measures the degree of ventilation inhomogeneity (VI). The test is used to identify small airway impairment in patients with lung diseases like cystic fibrosis. However, the physical and physiological factors that influence the test ou tcomes and differentiate health from disease are not well understood. Computational models have been used to better understand the interaction between anatomical structure and physiological properties of the lung, but none...
Source: PLoS Computational Biology - Category: Biology Authors: Source Type: research
Condition:   Bronchiectasis Adult Interventions:   Drug: Roflumilast;   Drug: Placebo oral tablet Sponsor:   University of Sao Paulo General Hospital Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Authors: Yoshie S, Omori K, Hazama A Abstract induced pluripotent stem (iPS) cells can be differentiated into various cell types, including airway epithelial cells, since they have the capacity for self-renewal and pluripotency. Thus, airway epithelial cells generated from iPS cells are expected to be potent candidates for use in airway regeneration and the treatment of airway diseases such as cystic fibrosis (CF). Recently, it was reported that iPS cells can be differentiated into airway epithelial cells according to the airway developmental process. These studies demonstrate that airway epithelial cells generated...
Source: Channels - Category: Molecular Biology Tags: Channels (Austin) Source Type: research
Publication date: 15 June 2019Source: New Scientist, Volume 242, Issue 3234Author(s): Clare Wilson
Source: New Scientist - Category: Science Source Type: research
We describe the use of transnasal humidified rapid-insufflation ventilatory exchange for deep sedation during dental extractions in a patient with severe cystic fibrosis as a precondition for lung transplantation. Patient wanted to have the procedure under general anesthesia with intubation due to extreme anxiety. However, we advocated deep IV sedation with transnasal humidified rapid-insufflation ventilatory exchange. Oxygenation was maintained without airway obstruction and pulmonary complications during the entire procedure of 110 minutes.
Source: A&A Case Reports - Category: Anesthesiology Tags: Case Reports Source Type: research
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Source: Acta Clinica Belgica - Category: General Medicine Authors: Source Type: research
The objectives of this study were to characterize the clinical outcomes and clearance of Mycobacterium abscessus complex (M. abscessus) from respiratory cultures in children with CF M. abscessus PD. Methods: This retrospective longitudinal cohort analysis evaluated the first course of treatment for M. abscessus PD in 33 children in Queensland, Australia between 2001 and 2015. Spirometry and nutritional outcomes 2 years pretreatment and 1 year posttreatment were compared with clearance or relapse/persistence of Mycobacterium abscessus complex from respiratory cultures. Results: Nine of 18 children who completed therap...
Source: The Pediatric Infectious Disease Journal - Category: Infectious Diseases Tags: Original Studies Source Type: research
AbstractThe past decade has witnessed a surge in research centered around exploring the role of the enigmatic innate immune-like lymphocyte MAIT cell in human disease. Recent evidence has led to the elucidation of its role as a potent defender at mucosal surfaces including lungs due to its capacity to mount a formidable immediate response to bacterial pathogens. MAIT cells have a unique attribute of recognizing microbial ligands in conjunction with non-classical MHC-related protein MR1. Recent studies have demonstrated their contribution in the pathogenesis of chronic pulmonary disorders including asthma and chronic obstru...
Source: Inflammation Research - Category: Research Source Type: research
ConclusionOur results highlight the importance of screening for large rearrangements in CF cases with no or only oneCFTR mutation defined.
Source: Molecular Genetics & Genomic Medicine - Category: Genetics & Stem Cells Authors: Tags: ORIGINAL ARTICLE Source Type: research
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