PubMed Journals Articles About "Scotland Provide Year Cystic Fibrosis Drug" RSS

19:02 EDT 23rd October 2019 | BioPortfolio

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Showing "Scotland provide year cystic fibrosis drug" PubMed Articles 1–25 of 30,000+

The presence of Aspergillus fumigatus is associated with worse respiratory quality of life in cystic fibrosis.

The clinical effects of Aspergillus fumigatus in the cystic fibrosis (CF) airway, with the exception of allergic bronchopulmonary aspergillosis, is unclear.

Hypertrophic osteoarthropathy presenting with joint pain in cystic fibrosis.

A 19 year old man with cystic fibrosis (CF) presented with pain and swelling of both wrists in association with an infective respiratory exacerbation. His CF-related complications included clubbing, multilobar bronchiectasis, chronic infection with Pseudomonas aeruginosa, exocrine pancreatic insufficiency, cystic fibrosis related diabetes mellitus, and low body mass. This article is protected by copyright. All rights reserved.

Oral non-steroidal anti-inflammatory drug therapy for lung disease in cystic fibrosis.

Progressive lung damage causes most deaths in cystic fibrosis. Non-steroidal anti-inflammatory drugs (such as ibuprofen) may prevent progressive pulmonary deterioration and morbidity in cystic fibrosis. This is an update of a previously published review.

Novel mutations and deletions in cystic fibrosis in a tertiary cystic fibrosis center in Istanbul.

Cystic fibrosis (CF) genotyping has garnered increased attention since the discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989 led to the identification of over 1700 mutations on chromosome 7. Yet, little is known about the genetic profile of CF patients in Turkey. This study sought to determine the mutation distribution among CF patients seeking care at Marmara University.

Deep learning to automate Brasfield chest radiographic scoring for cystic fibrosis.

The aim of this study was to evaluate the hypothesis that a deep convolutional neural network (DCNN) model could facilitate automated Brasfield scoring of chest radiographs (CXRs) for patients with cystic fibrosis (CF), performing similarly to a pediatric radiologist.

Clustered randomized controlled trial of a clinic-based problem-solving intervention to improve adherence in adolescents with cystic fibrosis.

In Cystic Fibrosis (CF), adherence to pulmonary medications is about 50% and decreases during adolescence. Effective interventions have not been integrated into CF care. This effectiveness study tested a brief, clinic-based behavioral intervention to improve adherence.

Singing as an adjunct therapy for children and adults with cystic fibrosis.

Cystic fibrosis is a genetically inherited, life-threatening condition that affects major organs. The management of cystic fibrosis involves a multi-faceted daily treatment regimen that includes airway clearance techniques, pancreatic enzymes and other medications. Previous studies have found that compliance with this intensive treatment is poor, especially among adolescents. Because of both the nature and consequences of the illness and the relentless demands of the treatment, many individuals with cystic ...

The role of geographical location and climate on recurrent Pseudomonas infection in young children with Cystic Fibrosis.

To determine the association between residence and climate with risk of Pseudomonas aeruginosa (Pa) and other respiratory outcomes.

Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.

Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent advent of the FDA-approved CFTR modulator drug ivacaftor, alone or in combination with lumacaftor or tezacaftor, has enabled treatment of the majority of patients suffering from CF. Even before the identification of the CFTR gene, gene therapy was put forward as a viable treatment option for this genetic condition. However, initial enthusiasm has be...

Acute Recurrent and Chronic Pancreatitis as Initial Manifestations of Cystic Fibrosis and Cystic Fibrosis Transmembrane Conductance Regulator-Related Disorders.

Recurrent pancreatitis is considered a rare manifestation of cystic fibrosis transmembrane conductance regulator (CFTR) dysfunction; this case series highlights that pancreatitis can be a presenting symptoms of cystic fibrosis (CF) or a CFTR-related disorder (CFTR-RD).

Successful adjunctive use of bacteriophage therapy for treatment of multidrug-resistant Pseudomonas aeruginosa infection in a cystic fibrosis patient.

We describe the use of bacteriophage therapy in a 26-year-old cystic fibrosis (CF) patient awaiting lung transplantation.

Abnormal pro-gly-pro pathway and airway neutrophilia in pediatric cystic fibrosis.

Proline-glycine-proline (PGP) is a bioactive fragment of collagen generated by the action of matrix metalloproteinase-9 (MMP-9) and prolylendopeptidase (PE), and capable of eliciting neutrophil chemotaxis and epithelial remodelling. PGP is normally then degraded by leukotriene A hydrolase (LTAH) to limit inflammation and remodelling. This study hypothesized that early and persistent airway neutrophilia in Cystic Fibrosis (CF) may relate to abnormalities in the PGP pathway and sought to understand underlying...

Mortality in Adults with Cystic Fibrosis Requiring Mechanical Ventilation: Cross-Sectional Analysis of Nationwide Events.

Survival in patients with cystic fibrosis (CF) is improving over time. Traditionally, there has been concern about high mortality in individuals with cystic fibrosis requiring invasive mechanical ventilation (IMV) for respiratory failure.

Non-fasting bioelectrical impedance analysis in cystic fibrosis: Implications for clinical practice and research.

Nutritional status affects pulmonary function in cystic fibrosis (CF) patients and can be monitored by using bioelectrical impedance analysis (BIA). BIA measurements are commonly performed in the fasting state, which is burdensome for patients. We investigated whether fasting is necessary for clinical practice and research.

Cystic fibrosis and insulin therapy: a reality check.

Diabetes is a complication of cystic fibrosis, caused by progressive loss of insulin secretion [1]. Insulin therapy seems logical for cystic fibrosis-related diabetes (CFRD) and is established in clinical practice. This article is protected by copyright. All rights reserved.

Impact of pharmacy services on cystic fibrosis transmembrane conductance regulator modulator prescribing at a pediatric cystic fibrosis center.

This study was undertaken to determine if the presence of a clinical pharmacy team impacted patients' access to cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

Lavage lipidomics signatures in children with cystic fibrosis and protracted bacterial bronchitis.

Balanced composition of a well-functioning pulmonary surfactant is crucial and essential for normal breathing. Here, we explored whether the composition of lipids recovered by broncho-alveolar lavage (BAL) in children with cystic fibrosis (CF) differ from children with protracted bacterial bronchitis (PBB) and controls. We wanted to differentiate, if alterations are primarily caused by the disease process or secondary due to an increased amount of cell-membrane lipids derived from inflammatory cells.

CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials.

Several treatment approaches in cystic fibrosis (CF) aim to correct CF transmembrane conductance regulator (CFTR) function; the efficacy of each approach is dependent on the mutation(s) present. A need remains for more effective treatments to correct functional deficits caused by the F508del mutation.

Correlating CFTR Function with Clinical Features to Inform Precision Treatment of Cystic Fibrosis.

The advent of precision treatment for cystic fibrosis (CF) using small-molecule therapeutics has created a need to estimate potential clinical improvements attributable to increases in cystic fibrosis transmembrane conductance regulator (CFTR) function.

Pharmacological analysis of CFTR variants of cystic fibrosis using stem cell-derived organoids.

Cystic fibrosis (CF) is a life-shortening genetic disease caused by mutations of CFTR, the gene encoding cystic fibrosis transmembrane conductance regulator. Despite considerable progress in CF therapies, targeting specific CFTR genotypes based on small molecules has been hindered because of the substantial genetic heterogeneity of CFTR mutations in patients with CF, which is difficult to assess by animal models in vivo. There are broadly four classes (e.g., II, III, and IV) of CF genotypes that differentia...

Cystic fibrosis screen positive inconclusive diagnosis (CFSPID): Experience in Tuscany, Italy.

The implementation of cystic fibrosis (CF) newborn screening (NBS) has led to identification of infants with a positive NBS test but inconclusive diagnosis classified as "CF screen positive, inconclusive diagnosis" (CFSPID). We retrospectively evaluated the prevalence and clinical outcome of CFSPID infants diagnosed by 2 NBS algorithms in the period from 2011 to 2016 in the Tuscany region of Italy.

Changes in Airway Microbiome and Inflammation with Ivacaftor Treatment in Patients with Cystic Fibrosis and the G551D Mutation.

Modulation of the cystic fibrosis transmembrane conductance regulator (CFTR) protein improves clinical outcomes in patients with cystic fibrosis (CF) and specific CFTR genetic mutations. It remains unclear how improving CFTR function modifies existing airway infection and inflammation.

Adherence to nutritional supplementation in cystic fibrosis.

The purpose of this study was to examine patterns of adherence to a novel dietary supplement in pediatric cystic fibrosis. Adherence to dietary supplementation in cystic fibrosis is challenging, and examination of patterns of adherence behavior over time is needed to better characterize subgroups of patients who need self-management support.

Mucin Binding To Therapeutic Molecules: The Case Of Antimicrobial Agents Used In Cystic Fibrosis.

Mucin is a complex glycoprotein consisting of a wide variety of functional groups that can interact with exogenous agents. The binding to mucin plays a crucial role in drug pharmacokinetics especially in diseases, such as cystic fibrosis (CF), where mucin is overexpressed. In this study, we have investigated the interaction between mucin and several drugs used in CF therapy. Protein-drug interaction was carried out by UV-Vis and fluorescence spectroscopy; quenching mechanism, binding constants, number of bi...

Gastrointestinal, Pancreatic, and Hepatic Manifestations of Cystic Fibrosis in the Newborn.

Gastrointestinal, pancreatic, and hepatic signs and symptoms represent the most common presentation of early disease among patients with cystic fibrosis and may be the initial indication of disease. Regardless of whether cystic fibrosis is diagnosed early by newborn screening or later by clinical course, the impact of gastrointestinal, pancreatic, and hepatic manifestations on early life is nearly ubiquitous. Conditions strongly linked with cystic fibrosis, such as meconium ileus and pancreatic insufficienc...

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