Results 51 to 60 of about 2,581 (157)

Clinical Use of Home Spirometry in Children With Cystic Fibrosis

Pediatric Pulmonology, Volume 61, Issue 6, June 2026.
ABSTRACT Background The use of home spirometry (HSPIR) has increased in pediatric cystic fibrosis (CF) care, but how it has been used clinically and its impact on clinical care have not been described. The purpose of this study was to address this knowledge gap through a secondary analysis of data from a HSPIR quality improvement project to ...
Lucy Tan, Andrew Borowiec, James A. Reed, Mark Rath, Reid Masi, Laura Bennett, Don B. Sanders, Daniel J. Weiner, Clement L. Ren   +8 more
wiley   +1 more source

Improved Quality of Life in Children With Cystic Fibrosis Who Received Transmembrane Conductance Regulator Modulators

Acta Paediatrica, Volume 115, Issue 6, Page 1283-1289, June 2026.
ABSTRACT Aim Children with cystic fibrosis (CF) face substantial daily treatment burdens and the effects of transmembrane conductance regulator modulators on these have not been sufficiently described. We evaluated changes in treatment burden after elexacaftor tezacaftor ivacaftor (ETI) was initiated.
Marcus Svedberg, Frida Lundqvist, Huda Abdulahi Östrand, Christina Krantz, Per Ekman, Henrik Imberg   +5 more
wiley   +1 more source

Challenges to Assessing the Prevalence of Cystic Fibrosis in the Caribbean

Pediatric Pulmonology, Volume 61, Issue 5, May 2026.
ABSTRACT Cystic fibrosis (CF) is likely underdiagnosed in Caribbean populations due to non‐representative cystic fibrosis transmembrane conductance regulator (CFTR) variant screening panels, limited newborn screening programs, and structural healthcare barriers.
Krystal L. Rivera‐Figueroa, Cole L. Martin, Christina A. Le, Wilfredo De Jesús Rojas, Leandra Cordero Oñate, Stephen G. Aller   +5 more
wiley   +1 more source

Female mice display sex-specific differences in cerebrovascular function and subarachnoid haemorrhage-induced injuryResearch in context

EBioMedicine
Summary: Background: In male mice, a circadian rhythm in myogenic reactivity influences the extent of brain injury following subarachnoid haemorrhage (SAH).
Danny D. Dinh, Hoyee Wan, Darcy Lidington, Steffen-Sebastian Bolz   +3 more
doaj   +1 more source

Systematic Review of Nasal Endoscopy Scores in Cystic Fibrosis Patients Treated With Cystic Fibrosis Transmembrane Conductance Regulator Modulators

Pediatric Pulmonology, Volume 61, Issue 4, April 2026.
ABSTRACT Introduction Cystic fibrosis (CF) is a severe genetic disorder caused by pathogenic variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, leading to multisystem complications including chronic rhinosinusitis and nasal polyposis. Recent advances in CFTR modulator therapies have revolutionized systemic disease control,
Rebecca Gallardo, Bruna Santos Silva, Lucas Sousa Salgado, José Dirceu Ribeiro, Eulalia Sakano   +4 more
wiley   +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

Orkambi® and amplifier co‐therapy improves function from a rare CFTR mutation in gene‐edited cells and patient tissue

EMBO Molecular Medicine, 2017
The combination therapy of lumacaftor and ivacaftor (Orkambi®) is approved for patients bearing the major cystic fibrosis (CF) mutation: ΔF508. It has been predicted that Orkambi® could treat patients with rarer mutations of similar “theratype”; however,
Steven V Molinski, Saumel Ahmadi, Wan Ip, Hong Ouyang, Adriana Villella, John P Miller, Po‐Shun Lee, Kethika Kulleperuma, Kai Du, Michelle Di Paola, Paul DW Eckford, Onofrio Laselva, Ling Jun Huan, Leigh Wellhauser, Ellen Li, Peter N Ray, Régis Pomès, Theo J Moraes, Tanja Gonska, Felix Ratjen, Christine E Bear   +20 more
doaj   +1 more source

Respiratory Oscillometry and Multimodal Lung Function Assessment of Elexacaftor/Tezacaftor/Ivacaftor Response in Children and Young Adults With Cystic Fibrosis

Pediatric Pulmonology, Volume 61, Issue 2, February 2026.
ABSTRACT Background To date, there is no data on forced oscillation technique (FOT) as a tool to evaluate elexacaftor/tezacaftor/ivacaftor (ETI) modulator in young patients with cystic fibrosis (pwCF). Objective Our study aimed to assess the effect of a 6‐month ETI treatment on FOT parameters and compare it to the effect on spirometry and nitrogen ...
Christos Kogias, Athina Sopiadou, Sotirios Fouzas, Petrina Vantsi, Elissavet‐Anna Chrysochoou, Evangelia Desli, Maria Galogavrou, Nikolaos Gasparis, Elpis Hatziagorou   +8 more
wiley   +1 more source

Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy

Drug Design, Development and Therapy, 2019
GJ ConnettNational Institute for Health Research, Southampton Respiratory Biomedical Research Centre, University Hospital Southampton NHS Foundation Trust, Southampton, SO16 6YD, UKAbstract: Lumacaftor-ivacaftor is a combination of two small molecule ...
Connett GJ
doaj  

Activity of lumacaftor is not conserved in zebrafish Cftr bearing the major cystic fibrosis‐causing mutation

FASEB BioAdvances, 2019
F508del‐cystic fibrosis transmembrane conductance regulator (CFTR) is the major mutant responsible for cystic fibrosis (CF). ORKAMBI®, approved for patients bearing this mutant, contains lumacaftor (VX‐809) that partially corrects F508del‐CFTR's ...
Onofrio Laselva, Steven Erwood, Kai Du, Zhenya Ivakine, Christine E. Bear   +4 more
doaj   +1 more source