Vertex Picks'Impressive' Cystic Fibrosis Drugs To Aim For A Multibillion-Dollar Market

Vertex Pharmaceuticals has picked two cystic fibrosis drugs to test in combination with its existing CF medicine, Kalydeco, as part of two different three-drug combinations aimed at helping as many as 90% of patients with cystic fibrosis. (Vertex's current offerings can treat half.)
Source: Healthcare News - Category: Pharmaceuticals Authors: Tags: NASDAQ:VRTX Source Type: news

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Over the past 20 years, there has been significant improvement in the treatment of cystic fibrosis (CF), which has prolonged survival from the second decade, well into the fourth decade of life. We sought to evaluate short, medium, and long-term outcomes of bronchial artery embolization (BAE) in this population.
Source: Journal of Vascular and Interventional Radiology : JVIR - Category: Radiology Authors: Tags: Scientific e-Posters Source Type: research
Publication date: Available online 21 February 2019Source: Journal of the Formosan Medical AssociationAuthor(s): Han-Sun Chiang, Ya-Yun Wang, Ying-Hung Lin, Yi-No WuCongenital bilateral absence of vas deferens (CBAVD) is a special entity in obstructive azoospermia. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene are involved in Taiwanese CBAVD but most heterozygous 5T variant. The solute carrier family 9 isoform 3 (SLC9A3) is the Na+/H+ exchanger, which interacts with CFTR and regulates the Ca2+ homeostasis. Loss of SLC9A3 decreases CFTR protein and causes obstructive azoospermia in mice. I...
Source: Journal of the Formosan Medical Association - Category: General Medicine Source Type: research
(Children's National Health System) Children and young adults with cystic fibrosis whose lung infections were treated with suboptimal doses of antibiotics had fewer changes in lung microbial diversity during the IV treatment, and their microbial diversity levels were higher 30 days later, a multi-institutional study that includes Children's researchers shows.
Source: EurekAlert! - Infectious and Emerging Diseases - Category: Infectious Diseases Source Type: news
Cell-based studies have shown that W1282X generates a truncated protein that can be functionally augmented by modulators. However, modulator treatment of primary cells from individuals who carry two copies of W1282X generates no functional CFTR. To understand the lack of response to modulators, we investigated the effect of W1282X on CFTR RNA transcript levels.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Original Article Source Type: research
CONCLUSIONS: We found that in patients with severe asthma, the prevalence of bronchiectasis is high and that bronchiectasis is associated with a longer asthma history, greater severity and, more importantly, chronic airflow obstruction. These findings are still insufficient evidence to considere features of asthma-bronchiectasis overlap syndrome, a distinct phenotype of severe asthma, but bronchiectasis is a frequent phenomenon leading to a more severe disease with frequent exacerbations. The performance of thorax HRCT on patients with severe asthma can help to evaluate management strategies for the disease in order to imp...
Source: Journal of Asthma - Category: Respiratory Medicine Tags: J Asthma Source Type: research
Condition:   Cystic Fibrosis Intervention:   Sponsor:   University Hospital, Montpellier Recruiting
Source: - Category: Research Source Type: clinical trials
AbstractCystic fibrosis (CF) is a progressive, genetic disease affecting multiple organ systems. Treatments are complex and take 2 –4 h per day. Adherence is 50% or less for pulmonary medications, airway clearance, and enzymes. Prior research has identified demographic and psychological variables associated with better adherence; however, no study has extensively identified facilitators of treatment adherence (e.g., adaptive behaviors and cognitions) in a sample of parents and adolescents. Forty-three participants were recruited from four CF centers as part of a larger measurement study. Participants included 29...
Source: Journal of Clinical Psychology in Medical Settings - Category: Psychiatry & Psychology Source Type: research
Airway infections are largely responsible for the morbidity and mortality of individuals with cystic fibrosis (CF). The CF microbiological landscape has undoubtedly shifted over the past two decades, with an increase in the recovery of filamentous fungi; Aspergillus fumigatus (Af) and Scedosporium species being the most prevalent [1,2]. In response, the CF community has raised several questions about the clinical significance of Af and other fungi detected in respiratory cultures, in the absence of an allergic host response.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Editorial Source Type: research
Biofabrication is a rapidly growing research field encompassing imaging, modelling, and printing technologies to produce personalised medical devices for patient anatomies [1,2]. Current cystic fibrosis (CF) treatment personalises physiotherapy and medications based on patient symptoms, genotype, and lifestyle [3]. Biofabrication could further individualise these treatments to CF's unique anatomical presentations, conveniently captured during routine medical imaging. For example, digital biofabrication models may prove useful to simulate and plan treatments, such as calculating dose and delivery of aerosol medications need...
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Editorial Source Type: research
Publication date: Available online 20 February 2019Source: Canadian Journal of DiabetesAuthor(s): Julie Gilmour
Source: Canadian Journal of Diabetes - Category: Endocrinology Source Type: research
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