Vertex’s cystic fibrosis treatment makes it a hot company

Biotech group’s promising results help it defy broader investor fears over sector
Source: - Drugs and Healthcare - Category: Pharmaceuticals Source Type: news

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Publication date: Available online 20 July 2018Source: The Lancet Respiratory MedicineAuthor(s): Barry Linnane, Paul McNally
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
Publication date: Available online 20 July 2018Source: The Lancet Respiratory MedicineAuthor(s): Julian T Forton, Iolo Doull, Lena P Thia, CF-SpIT authors
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
The Q359K/T360K mutation, described in Jewish CF patients of Georgian decent, is of questionable clinical significance.
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Tags: Short Communication Source Type: research
Autosomal dominant polycystic kidney disease (ADPKD) is associated with progressive enlargement of cysts, leading to a decline in function and renal failure that cannot be prevented by current treatments. Mutations in pkd1 and pkd2, encoding the polycystin 1 and 2 proteins, induce growth-related pathways, including heat shock proteins, as occurs in some cancers, raising the prospect that pharmacological interventions that target these pathways might alleviate or prevent ADPKD. Here, we demonstrate a role for VX-809, a corrector of cystic fibrosis transmembrane conductance regulator (CFTR), conventionally used to manage cys...
Source: Journal of Biological Chemistry - Category: Chemistry Authors: Tags: Molecular Bases of Disease Source Type: research
Authors: Ratchford TL, Teckman JH, Patel DR Abstract INTRODUCTION: Cystic fibrosis (CF) is a severe, progressive, multi-systemic disease that is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Optimizing nutrition is critical, as higher growth parameters are associated with better pulmonary function and outcomes, but unfortunately patients with this disease are prone to malnutrition, growth failure, and vitamin deficiencies. The purpose of this review is to provide a timely highlight of the physiologic processes and outcome data to support today's management strategies, a...
Source: Expert Review of Gastroenterology and Hepatology - Category: Gastroenterology Tags: Expert Rev Gastroenterol Hepatol Source Type: research
Publication date: Available online 18 July 2018Source: Medical Mycology Case ReportsAuthor(s): Tjomme van der Bruggen, Anna Kolecka, Bart Theelen, Johanna M. Kwakkel-van Erp, Bert Arets, Teun BoekhoutAbstractCystic fibrosis (CF) patients are colonized with a multitude of bacteria and fungi. From respiratory samples of two CF patients in our institute, a difficult to identify yeast was isolated repeatedly. This yeast was eventually identified as Cutaneotrichosporon (Cryptococcus) cyanovorans by internal transcribed spacer (ITS) and ribosomal large subunit (LSU) sequencing. C. cyanovorans is a basidiomycetous yeast originall...
Source: Medical Mycology Case Reports - Category: Biology Source Type: research
A 39-year-old non-smoker woman was admitted in the thoracic oncology unit of the Montpellier Academic Hospital in June 2017 for investigation of multiple lung consolidations, fever, and respiratory failure. She was followed-up since infancy for a cystic fibrosis with cystic fibrosis transmembrane conductance regulator (CFTR) mutation ( ΔF508-CFTR). Two years ago, a lung allograft was decided, owing to rapid onset of respiratory function impairment, and she received a bi-pulmonary transplant. According to the donor database, the lung transplant was removed from a 57-year-old woman with a 30 pack-year smoking history.
Source: Lung Cancer - Category: Cancer & Oncology Authors: Source Type: research
Pediatric Diabetes,Volume 0, Issue ja, -Not available-.
Source: Pediatric Diabetes - Category: Endocrinology Authors: Source Type: research
CONCLUSIONS: Pilates should be considered for patients with NCDs, as it improves exercise tolerance. Future studies with robust methodologies are still needed to clarify its effectiveness on outcomes with moderate, limited or conflicting evidence and to establish the most suitable intervention protocol. PMID: 30012382 [PubMed - in process]
Source: Complementary Therapies in Medicine - Category: Complementary Medicine Authors: Tags: Complement Ther Med Source Type: research
Antibiotics suffer from eventual resistance by the bacteria and the fact that they can be quite indiscriminate in who they attack, including the gut’s healthy microbiome. But, there are also bacteriophages, which are viruses that can kill spec...
Source: Medgadget - Category: Medical Devices Authors: Tags: Medicine Nanomedicine Source Type: blogs
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