Results 51 to 60 of about 4,639 (201)

Impact of lumacaftor/ivacaftor on the bacterial and fungal respiratory pathogens in cystic fibrosis: a prospective multicenter cohort study in Sweden

Therapeutic Advances in Respiratory Disease
Background: A significant decline in pulmonary exacerbation rates has been reported in CF patients homozygous for F508del treated with lumacaftor/ivacaftor. However, it is still unclear whether this reduction reflects a diminished microbiological burden.
Mahasin Al Shakirchi, Kimmo Sorjonen, Lena Hjelte, Lena Klingspor, Peter Bergman, Petrea Ericson, Marcus Svedberg, Ulrika Lindberg, Christine Hansen, Isabelle de Monestrol   +9 more
doaj   +1 more source

Lumacaftor/ivacaftor initiation in two liver transplantation patients under tacrolimus and antifungal azoles

Clinical Case Reports, 2019
Key Clinical Message We report the initiation of CFTR modulator lumacaftor/ivacaftor combination (LUM/IVA) in two adolescents with cystic fibrosis who were treated with antifungal azoles (AZO) and tacrolimus (TCS) for liver transplantation.
Ikrame Chouchane, Nathalie Stremler‐Lebel, Philippe Reix   +2 more
doaj   +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

Chronic inflammation in CF airways - a persistent issue for A20 [PDF]

, 2016
Cystic Fibrosis (CF) is characterised by prolonged and exaggerated airways inflammation. Despite recent developments to overcome the underlying functional defect in CFTR (cystic fibrosis transmembrane conductance regulator), there is still an unmet need ...
El Banna, Amal, Ennis, Madeleine, McCollum, Kirsten, Schock, Bettina   +3 more
core   +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

Cost-effectiveness analysis of lumacaftor and ivacaftor combination for the treatment of patients with cystic fibrosis in the United States

Orphanet Journal of Rare Diseases, 2018
Background Lumacaftor/ivacaftor was approved by the Food and Drug Administration (FDA) as a combination treatment for Cystic Fibrosis (CF) patients who are homozygous for the F508del mutation.
Dolly Sharma, Shan Xing, Yu-Ting Hung, Rachel N. Caskey, Maria L. Dowell, Daniel R. Touchette   +5 more
doaj   +1 more source

CFTR Modulators Dampen Aspergillus-Induced Reactive Oxygen Species Production by Cystic Fibrosis Phagocytes

Frontiers in Cellular and Infection Microbiology, 2020
Excessive inflammation by phagocytes during Aspergillus fumigatus infection is thought to promote lung function decline in CF patients. CFTR modulators have been shown to reduce A.
Alexander J. Currie, Alexander J. Currie, Ellen T. Main, Heather M. Wilson, Darius Armstrong-James, Adilia Warris   +5 more
doaj   +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

Safety, Tolerability, and Effects of Sodium Bicarbonate Inhalation in Cystic Fibrosis. [PDF]

, 2020
BACKGROUND:Among the many consequences of loss of CFTR protein function, a significant reduction of the secretion of bicarbonate (HCO3-) in cystic fibrosis (CF) is a major pathogenic feature.
Clinckspoor, Karl Jan, Conrad, Douglas, Gomez, Carla Cristina Souza, Levy, Carlos Emilio, Marson, Fernando Augusto Lima, Mauch, Renan Marrichi, Parazzi, Paloma Lopes Francisco, Peixoto, Andressa Oliveira, Pessine, Francisco Benedito Teixeira, Quinton, Paul Marquis, Ribeiro, Antônio Fernando, Ribeiro, José Dirceu, Ribeiro, Maria Ângela Gonçalves Oliveira   +12 more
core   +1 more source

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