Results 71 to 80 of about 9,635 (207)

Health state utility data in Cystic Fibrosis: A systematic review [PDF]

, 2019
Introduction: Cystic fibrosis (CF) is a life-limiting, hereditable condition, with the highest prevalence in Europe. CF treatments have led to improvements in clinical symptoms, disease management and decelerated disease progression.
Archangelidi, Olga, Bhadhuri, Arjun, Bilton, Diana, Carr, Siobhan, Mohindru, Bishal, Sach, Tracey, Turner, David, Whitty, Jennifer   +7 more
core   +1 more source

3D Automated Segmentation of Bronchial Abnormalities on Ultrashort Echo Time MRI: A Quantitative MR Outcome in Cystic Fibrosis

Journal of Magnetic Resonance Imaging, Volume 63, Issue 4, Page 1190-1200, April 2026.
ABSTRACT Background Cystic fibrosis (CF) monitoring relies on computed tomography (CT), but ultra‐short echo time MRI (UTE‐MRI) offers a radiation‐free alternative. However, its clinical adoption is hindered by the laborious and subjective manual analysis, which prevents standardized quantification of bronchial abnormalities.
Amel Imene Hadj Bouzid, Ilyes Benlala, Baudouin Denis de Senneville, Fabien Baldacci, Julie Macey, Wadie Benhassen, Pan Su, Stephanie Bui, Maeva Zysman, Aurelien Bustin, Patrick Berger, Gael Dournes   +11 more
wiley   +1 more source

A treatment evaluator tool to monitor the real-world effectiveness of inhaled aztreonam lysine in cystic fibrosis [PDF]

, 2017
Background: Studies are required that evaluate real-world outcomes of inhaled aztreonam lysine in patients with cystic fibrosis (CF). Methods: Our treatment-evaluator tool assessed the effectiveness of inhaled aztreonam in routine practice in 117 CF ...
Amadori, Andrew M Jones, Assael, Barry, Barry J Plant, Bell, Britto, Cano Megías, Cedric Gunaratnam, Charles S Haworth, Cherniak, Damian G Downey, Daniel G Peckham, Davies, de Boer, Diana Bilton, Döring, Edward F McKone, Flume, Garrison, Harrison, Herland, Joe A Eustace, Kerem, Liou, McCoy, McKone, Mogayzel, National Research Ethics Service (NRES), Oermann, Plant, Quittner, R. Ian Ketchell, Retsch-Bogart, Roche, Sanders, Steinkamp   +36 more
core   +1 more source

The Race to Salvage Glucocerebrosidase: Understanding Small‐Molecule Therapies for GBA1‐Associated Parkinsonism

Movement Disorders, Volume 41, Issue 3, Page 596-602, March 2026.
Variants in GBA1, the gene encoding the lysosomal enzyme glucocerebrosidase, cause Gaucher disease and confer an increased risk for parkinsonism. Strategies using small molecules can improve the function of glucocerebrosidase in lysosomes. A clear understanding of the mechanism‐of‐action of these compounds will facilitate development of GBA1‐modulating
Mark J. Henderson, Tiffany C. Chen, Logan M. Glasstetter, Yu Chen, Juan J. Marugan, Ellen Sidransky   +5 more
wiley   +1 more source

Assessing Dietary Patterns and Composition Among Adults With Cystic Fibrosis Taking Highly Effective Modulator Therapy

Pediatric Pulmonology, Volume 61, Issue 3, March 2026.
ABSTRACT Background The metabolic impact of poor diet quality in cystic fibrosis (CF), coupled with a rise in obesity and modulator‐induced weight gain, is a growing concern. Our study aimed to understand knowledge and perspectives regarding dietary changes on modulators, and how measured nutrient intake changes with different dietary patterns in ...
Julianna Bailey, Natalie R. Rose, Ashritha R. Chalamalla, Justin D. Anderson, Elizabeth Baker, Jennifer S. Guimbellot   +5 more
wiley   +1 more source

Newborn screening for cystic fibrosis [PDF]

, 2016
Since the late 1970s when the potential of the immunoreactive trypsinogen assay for early identification of infants with cystic fibrosis was first recognised, the performance of newborn blood spot screening (NBS) has been continually assessed and its use
Castellani, C, Massie, J, Sontag, M, Southern, KW   +3 more
core   +1 more source

Governments need better guidance to maximise value for money: the case of Australia’s Pharmaceutical Benefits Advisory Committee [PDF]

, 2016
Published online: 27 January 2016In Australia, the Pharmaceutical Benefits Advisory Committee (PBAC) makes recommendations to the Minister for Health on which pharmaceuticals should be subsidised.
Carter, D.A., Haji Ali Afzali, H., Vogan, A.   +2 more
core   +1 more source

Disparities in Access to Cystic Fibrosis Therapy Across Countries

Pediatric Pulmonology, Volume 61, Issue 3, March 2026.
ABSTRACT Cystic fibrosis has been transformed by the development of CFTR modulator therapies, with substantial improvements in survival and quality of life. However, access to these therapies remains profoundly unequal worldwide. The greatest benefits have been realized in high‐income countries, while people with cystic fibrosis in low‐ and middle ...
Bulent Karadag
wiley   +1 more source

Role of the SLC26A9 chloride channel as disease modifier and potential therapeutic target in cystic fibrosis [PDF]

, 2018
The solute carrier family 26, member 9 (SLC26A9) is an epithelial chloride channel that is expressed in several organs affected in patients with cystic fibrosis (CF) including the lungs, the pancreas, and the intestine. Emerging evidence suggests SLC26A9
Balázs, Anita, Mall, Marcus A.
core   +1 more source

Open Systems Pharmacology Community Conference (OSP‐CC) Proceedings 2025

CPT: Pharmacometrics &Systems Pharmacology, Volume 15, Issue 3, March 2026.
ABSTRACT Developed at Bayer Technology Services, PK‐Sim and MoBi transitioned into the Open Systems Pharmacology (OSP) Suite, released as free open‐source software in 2017. An active community with stakeholders from academia, industries, and regulators contributes to the continuous improvement of open‐source model‐informed drug development (MIDD). This
Henrik Cordes, Pieter Annaert, Pavel Balazki, Salih Benamara, Rodolfo Hernandes Bonan, Andrew Butler, Marylore Chenel, Yunhai Cui, André Dallmann, Wilhelmus E. A. de Witte, Cleo Demeester, Denise Feick, Florence Gattacceca, René Geci, Grégori Gerebtzoff, Andrea Gruber, Mariana Guimarães, Abdullah Hamadeh, Nina Hanke, Manuel Ibarra, Ibrahim Ince, Tobias Kanacher, Lars Kuepfer, Felix Mil, Ghazal Montaseri, Nina Nauwelaerts, Susana Proenca, Stephan Schaller, Jan Frederik Schlender, Annika R. P. Schneider, Erik Sjögren, Juri Solodenko, Alexander Staab, Paul Vrenken, Carla Troisi, Donato Teutonico   +35 more
wiley   +1 more source