Results 51 to 60 of about 9,288 (216)

Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis

Health Technology Assessment, 2014
Background: Ivacaftor (Kalydeco®, Vertex Pharmaceuticals) is the first of a new class of drugs that target the underlying protein defect in cystic fibrosis (CF).
Penny Whiting, Maiwenn Al, Laura Burgers, Marie Westwood, Steve Ryder, Martine Hoogendoorn, Nigel Armstrong, Alex Allen, Hans Severens, Jos Kleijnen   +9 more
doaj   +1 more source

Adapted Linear Binning Method to Assess Pulmonary Ventilation and Perfusion in Children Using Lung Matrix Pencil Decomposition (MP)‐MRI

Magnetic Resonance in Medicine, EarlyView.
ABSTRACT Purpose To adapt and validate a linear binning technique, developed for hyper‐polarized 129Xe MRI, for functional lung MRI with matrix‐pencil decomposition (MP)‐MRI. Methods First, a dedicated normalization was applied to the perfusion‐weighted and the ventilation‐weighted map histograms.
Marion Curdy, Carmen Streibel, Oliver Bieri, Elisabeth Kieninger, Florian Wyler, Lukas Ebner, Grzegorz Bauman, Philipp Latzin   +7 more
wiley   +1 more source

Real-world effectiveness of ivacaftor in children with cystic fibrosis and the G551D mutation

Anales de Pediatría (English Edition), 2019
Introduction: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has been shown to improve the nutritional status and lung function of cystic fibrosis patients with the G551D mutation in clinical trials.
David Gomez-Pastrana, Chinedu Nwokoro, Mike McLean, Sarah Brown, Nanna Christiansen, Caroline S. Pao   +5 more
doaj   +1 more source

Elexacaftor/tezacaftor/ivacaftor’s effects on cystic fibrosis infections are maintained, but not increased, after 3.5 years of treatment

Journal of Clinical Investigation
To the Editor: Elexacaftor/tezacaftor/ivacaftor (ETI) is the most effective modulator drug currently available for most people with cystic fibrosis (PwCF) (1).
Sarah J. Morgan, E. Coulter, Hannah L Betts, George M. Solomon, J. P. Clancy, Steven M. Rowe, David P. Nichols, Pradeep K. Singh   +7 more
semanticscholar   +1 more source

Updated EAACI Statement on Drug Hypersensitivity Skin Testing: Methodology and Non‐Irritative Concentrations

Allergy, EarlyView.
ABSTRACT These updated EAACI guidelines aim to standardize skin testing methodologies for both immediate and non‐immediate drug hypersensitivity reactions. For immediate reactions, the optimal testing window is 4–6 weeks post‐reaction; whereas beyond 6 months, false‐negative results increase.
Annick Barbaud, Lene Heise Garvey, Elizabeth Phillips, Michael Ardern‐Jones, Sevim Bavbek, Patrizia Bonadonna, Jean‐Christoph Caubet, Ana‐Maria Copaescu, Valentina Gueli, Jose Julio Laguna, Ricardo Madrigal‐Burgaleta, Francesca Mori, Vito Sabato, Maria J Torres, Knut Brockow   +14 more
wiley   +1 more source

The use of targeted therapy lumacaftor/ivacaftor in patients with cystic fibrosis

Медицинский совет, 2022
Accepted, basic therapy of cystic fibrosis (CF), until recently, was symptomatic and aimed at slowing down pathological processes, mainly from the respiratory system and gastrointestinal tract, caused by a defect in the CFTR gene.
A. G. Chermensky, T. E. Gembitskaya, A. V. Orlov, V. R. Makhmutova   +3 more
doaj   +1 more source

Impact of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in children with cystic fibrosis and one or two F508del alleles

European Respiratory Journal
Extract Clinical trials of the triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI) demonstrated unprecedented clinical benefits including improvements in lung function ...
M. Stahl, M. Dohna, S. Graeber, Olaf Sommerburg, D. Renz, S. Pallenberg, A. Voskrebenzev, K. Schütz, G. Hansen, F. Doellinger, E. Steinke, Stephanie Thee, Jobst Röhmel, S. Barth, C. Rückes-Nilges, J. Berges, S. Hämmerling, M. Wielpütz, L. Naehrlich, J. Vogel-Claussen, B. Tümmler, M. Mall, Anna-Maria Dittrich   +22 more
semanticscholar   +1 more source

Cystic Fibrosis and Colorectal Cancer Risk: Reprogramming of the Intestinal Epithelial Niche and Cell‐State Plasticity in the CFTR Modulator Era

Cell Proliferation, EarlyView.
In cystic fibrosis disorder, CFTR dysfunction drives multilayered intestinal niche disruption, including dysbiosis, inflammation, hypoxia, metabolic stress and genotoxic injury, promoting cell‐state plasticity and a pre‐neoplastic ecosystem. CFTR modulators and environmental factors may partially reshape these processes, highlighting opportunities for ...
Bala Umashankar, Ines Pankonien, Margarida Amaral, Chee Y. Ooi, Thierry Jardé, Shafagh A. Waters   +5 more
wiley   +1 more source

Physiologically‐based pharmacokinetic modeling of the drug–drug interaction between ivacaftor and lefamulin in cystic fibrosis patients

CPT: Pharmacometrics & Systems Pharmacology
Lefamulin is being evaluated as a treatment for bacterial exacerbations in cystic fibrosis (CF). Ivacaftor is approved for the treatment of patients with CF.
Magali Amiel, Alice Ke, Steven P. Gelone, Hannah M. Jones, Wolfgang Wicha   +4 more
doaj   +1 more source

The effect of CFTR modulators on a cystic fibrosis patient presenting with recurrent pancreatitis in the absence of respiratory symptoms: a case report

BMC Gastroenterology, 2019
Background Cystic fibrosis (CF) is a genetic disorder of the epithelial CFTR apical chloride channel resulting in multi-organ manifestations, including pancreatic exocrine secretion.
J. Dixon Johns, Steven M. Rowe
doaj   +1 more source