Results 61 to 70 of about 12,467 (245)

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

Journal of Magnetic Resonance Imaging, EarlyView.
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

Comparison of Single‐Breath and Multi‐Breath Xe‐MRI in the Longitudinal Assessment of Treatment in Children With Cystic Fibrosis

Journal of Magnetic Resonance Imaging, EarlyView.
ABSTRACT Background Elexacaftor/tezacaftor/ivacaftor (ETI) is a current standard therapy for pediatric cystic fibrosis (CF). Multiple‐breath washout 129Xe MRI (MBW Xe‐MRI) is improved following 1 month of treatment. However, the utility of MBW Xe‐MRI over extended ETI treatment and its comparison to single‐breath Xe‐MRI and pulmonary function tests ...
Faiyza S. Alam, Samal Munidasa, Brandon Zanette, Daniel Li, Sharon Braganza, Felix Ratjen, Giles Santyr   +6 more
wiley   +1 more source

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

Cystic fibrosis modulator therapy can reverse cystic bronchiectasis

Respirology Case Reports, 2023
Bronchiectasis is often considered progressive and irreversible, so cases of regression or reversal are an important step in understanding the underlying pathophysiological mechanisms.
Peter G. Middleton, Nicholas J. Simmonds
doaj   +1 more source

Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes

Journal of Clinical & Translational Endocrinology, 2023
Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin, Sophie Gohy, Michel P. Hermans, Vanessa Preumont   +3 more
doaj   +1 more source

Elexacaftor–Tezacaftor–Ivacaftor Reduces Revision Sinus Surgery in People With Cystic Fibrosis

The Laryngoscope, EarlyView.
Elexacaftor–tezacaftor–ivacaftor (ETI) was associated with a significant decrease in the frequency and rate of endoscopic sinus surgeries in patients with cystic fibrosis. These findings suggest an improvement in CF‐related chronic rhinosinusitis outcomes following ETI and may influence future CF treatment decisions.
Amy Lin, Makanko Komara, Isaac Kistler, Zheng Hong Tan, Meredith Lind, Kavitha Kotha, Tendy Chiang   +6 more
wiley   +1 more source

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

Movement Disorders, EarlyView.
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

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

Children, 2023
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic ...
Nikoletta Kapouni, Maria Moustaki, Konstantinos Douros, Ioanna Loukou   +3 more
doaj   +1 more source

Obstetric and Neonatal Outcomes in Pregnancies From a Dedicated Cystic Fibrosis‐Maternal Health Service: A Retrospective Study

BJOG: An International Journal of Obstetrics &Gynaecology, EarlyView.
ABSTRACT Objective A comprehensive review of maternal, obstetric and neonatal outcomes in pregnancies in females with cystic fibrosis (fwCF) following the introduction of Elexacaftor/Tezacaftor/Ivacaftor (ETI) therapy in a novel, dedicated CF‐Maternal Health service.
Rebecca Scott, Amy Downes, Ladina Weitnauer, Rebecca Dobra, Natasha Singh, Rachel Robinson, Emily Diable, Saraha Collins, Elaine Bowman, Thomas Tobin, Andrew Jones, Nicholas Simmonds, Imogen Felton   +12 more
wiley   +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