FDA Approves New Cystic Fibrosis Drug

The treatment, Trikafta, increases lung function in most patients with the disease—but comes with a hefty price tag.
Source: The Scientist - Category: Science Tags: News & Opinion Source Type: news

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Bronchiectasis is a complex, chronic respiratory condition, characterized by frequent cough and exertional dyspnea due to a range of conditions that include inherited mucociliary defects, inhalational airway injury, immunodeficiency states and prior respiratory infections. For years, bronchiectasis was classified as either being caused by cystic fibrosis or non-cystic fibrosis. Non-cystic fibrosis bronchiectasis, once considered an orphan disease, is more prevalent worldwide in part due to greater availability of chest computed tomographic imaging.
Source: Respiratory Medicine - Category: Respiratory Medicine Authors: Tags: Review article Source Type: research
Publication date: April 2020Source: Stem Cell Research, Volume 44Author(s): Sylvia Merkert, Madline Schubert, Alexandra Haase, Hettie M. Janssens, Bob Scholte, Nico Lachmann, Gudrun Göhring, Ulrich Martin
Source: Stem Cell Research - Category: Stem Cells Source Type: research
L. Sinn Extracellular vesicles (EVs) are a class of naturally occurring secreted cellular bodies that are involved in long distance cell-to-cell communication. Proteins, lipids, mRNA, and miRNA can be packaged into these vesicles and released from the cell. This information is then delivered to target cells. Since EVs are naturally adapted molecular messengers, they have emerged as an innovative, inexpensive, and robust method to deliver therapeutic cargo in vitro and in vivo. Well-differentiated primary cultures of human airway epithelial cells (HAE) are refractory to standard transfection techniques. Indeed, common s...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Article Source Type: research
Abstract Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation. While the hope is that these drug compounds will prevent lung damage when started early in life, there is an ongoing need to care for people with advanced lung disease. The focus of this review is the accumulating data from clinical trials and case series regarding the benefits of CFTR modulator therapy in people with advanced pulmonary disease. We address ...
Source: Respiratory Care - Category: Respiratory Medicine Authors: Tags: Eur Respir Rev Source Type: research
Stenotrophomonas maltophilia is a Gram-negative bacterium found ubiquitously in the environment that has historically been regarded as nonpathogenic. S. maltophilia is increasingly observed in patient sputa in cystic fibrosis (CF), and while existing epidemiology indicates that patients with S. maltophilia have poorer diagnoses, its clinical significance remains unclear. Moreover, as multidrug resistance is common among S. maltophilia isolates, treatment options for these infections may be limited. Here, we investigated the pathogenicity of S. maltophilia alone and during polymicrobial infection with Pseudomonas aeruginosa...
Source: Infection and Immunity - Category: Infectious Diseases Authors: Tags: Host-Associated Microbial Communities Source Type: research
AbstractATP-binding cassette (ABC) transporters constitute a superfamily of 48 structurally similar membrane transporters that mediate the ATP-dependent cellular export of a plethora of endogenous and xenobiotic substances. Importantly, genetic variants inABC genes that affect gene function have clinically important effects on drug disposition and can be predictors of the risk of adverse drug reactions and efficacy of chemotherapeutics, calcium channel blockers, and protease inhibitors. Furthermore, loss-of-function of ABC transporters is associated with a variety of congenital disorders. Despite their clinical importance,...
Source: Human Genetics - Category: Genetics & Stem Cells Source Type: research
Cystic fibrosis (CF) is characterized by chronic respiratory infection leading to severe lung degradation [1,2]. The pathological mucus in the lungs of CF patients has altered biophysical properties (e.g., viscosity, elasticity) and results in decreased or failed mucociliary clearance, impairment of the immune response, and decreased antibiotic efficacy [3 –5]. The altered biophysical characteristics of CF mucus are due in large part to elevated concentrations of macromolecules (e.g., mucins, DNA) and decreased volume of the airway surface liquid [2,4–6].
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Source Type: research
Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation. While the hope is that these drug compounds will prevent lung damage when started early in life, there is an ongoing need to care for people with advanced lung disease. The focus of this review is the accumulating data from clinical trials and case series regarding the benefits of CFTR modulator therapy in people with advanced pulmonary disease. We address the impact of trea...
Source: European Respiratory Review - Category: Respiratory Medicine Authors: Tags: CF and non-CF bronchiectasis Reviews Source Type: research
In the past 10 years the incremental success of modulators of the cystic fibrosis transmembrane regulator (CFTR) protein have improved outcomes for people with cystic fibrosis (CF) [1]. Ivacaftor for individuals with sequence variants associated with class III mutations (e.g. G551D) and some other residual function mutations of the CFTR gene followed by lumacaftor/ivacaftor or tezacaftor/ivacaftor combinations in individuals homozygous for the F508del have achieved improvements in quality of life reduction, pulmonary exacerbations and variable improvement in forced expiratory volume in 1 s (FEV1). Highly effectiv...
Source: European Respiratory Review - Category: Respiratory Medicine Authors: Tags: CF and non-CF bronchiectasis Editorial Source Type: research
Abstract The Stenotrophomonas maltophilia complex (Smc) comprises opportunistic environmental Gram negative bacilli responsible for a variety of infections in both humans and animals. Beyond its large genetic diversity, its genetic organization in genogroups was recently confirmed through the whole genome sequencing of human and environmental strains. Animal strains being poorly represented in these analyses, we sequenced the whole genomes of 93 animal strains to determine their genetic background and characteristics. Combining these data with 81 newly sequenced human strains and the genomes available from RefSeq,...
Source: Applied and Environmental Microbiology - Category: Microbiology Authors: Tags: Appl Environ Microbiol Source Type: research
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