Results 1 to 10 of about 89 (70)

The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis [PDF]

Expert Opinion on Drug Safety, 2017
Lumacaftor-ivacaftor is indicated for treatment of cystic fibrosis (CF) in patients homozygous for the Phe-508del cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations. In clinical trials, treated patients showed improved pulmonary function, reduced pulmonary exacerbations, and other benefits.
Susanna A Mccolley
exaly   +3 more sources

Spectral Analysis on Cuba-Lumacaftor: Cubane as Benzene Bioisosteres of Lumacaftor

ACS Omega, 2023
In this paper, we theoretically investigate the electronic structure and physical properties of cuba-lumacaftor, cubane as benzene bioisosteres of lumacaftor, stimulated by recent experimental reports [Wiesenfeldt M. P.; Nature2023, 618, 513-518]. The permanent electric dipole moments of cuba-lumacaftor in neutral, acidic, and alkaline environments are
Dongdong Wang, Xiaohong Lyu, Mengtao Sun, Yongqiang Liang   +3 more
openaire   +3 more sources

Bridging for lung transplantation with lumacaftor/ivacaftor [PDF]

Breathe, 2018
The case of a young female cystic fibrosis patient, homozygous for delta F508 and with terminal respiratory insufficiency, who started treatment with lumacaftor/ivacaftor 
http://ow.ly/4I0t30kftDx.
Pedersen, Søren Sperling, Jensen-Fangel, Søren, Jeppesen, Majbritt, Søgaard, Ole Schmeltz   +3 more
openaire   +4 more sources

Long‐term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real‐world cohort study

British Journal of Clinical Pharmacology, Volume 92, Issue 6, Page 1822-1832, June 2026.
Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Nicola Perrotta, Luigi Angelo Fiorito, Roberta Vescovo, Anna Virgilio, Giulia Amato, Giuseppe Cimino   +5 more
wiley   +1 more source

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

Utility of Fecal Elastase‐1 in Estimating Exocrine Pancreatic Function in Cystic Fibrosis: A Scoping Review

Pediatric Pulmonology, Volume 61, Issue 5, May 2026.
ABSTRACT Background In people with cystic fibrosis (pwCF), identification of exocrine pancreatic insufficiency (EPI) is essential to prevent steatorrhea and, if not managed actively, can lead to catastrophic consequences. Fecal elastase‐1 (FE‐1) is a widely used test to screen for EPI in cystic fibrosis (CF).
Senthilkumar Sankararaman, Sara Hendrix, Mandy Neudecker, Drucy Borowitz   +3 more
wiley   +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