Results 41 to 50 of about 2,330 (177)

Elexacaftor–Tezacaftor–Ivacaftor improves exercise capacity in adolescents with cystic fibrosis [PDF]

Pediatric Pulmonology, 2022
AbstractObjectiveElexacaftor/Tezacaftor/Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator with the potential to improve exercise capacity. This case series of three adolescents with CF aimed to investigate whether 6 weeks treatment with Elexacaftor/Tezacaftor/Ivacaftor could improve exercise capacity in CFTR modulator ...
Adam J. Causer, Janis K. Shute, Michael H. Cummings, Anthony I. Shepherd, Samuel R. Wallbanks, Richard M. Pulsford, Victoria Bright, Gary Connett, Zoe L. Saynor   +8 more
openaire   +3 more sources

USO DE EXACAFTOR/ TEZACAFTOR /IVACAFTOR EN PACIENTES CON FIBROSIS QUÍSTICA POST-TRASPLANTE PULMONAR

Neumología Pediátrica, 2023
El uso de moduladores de CFTR en pacientes con fibrosis quística post trasplante pulmonar es un tema todavía controversial. Varias publicaciones reportan los beneficios del modulador elexacaftor/tezacaftor/ivacaftor en los síntomas extrapulmonares de la ...
Pablo Jorquera Pinto
doaj   +1 more source

Successful pregnancy in a cystic fibrosis patient with a severe impairment of lung function receiving Elexacaftor-Tezacaftor-Ivacaftor

Respiratory Medicine Case Reports, 2022
Before the arrival of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators women with CF and impaired lung function were experiencing a high risk of complications and mortality during and the years after pregnancy.
Zisis Balmpouzis, Annabelle Faure van Rossum, David Baud, Alice Panchaud, Georgia Mitropoulou, Jesica Mazza Stalder, Angela Koutsokera   +6 more
doaj   +1 more source

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

New England Journal of Medicine, 2019
Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del CFTR mutation. In a phase 2 trial involving patients who were heterozygous for the Phe508del CFTR mutation and a minimal-function mutation (Phe508del ...
Middleton P. G., Mall M. A., Drevinek P., Lands L. C., McKone E. F., Polineni D., Ramsey B. W., Taylor-Cousar J. L., Tullis E., Vermeulen F., Marigowda G., McKee C. M., Moskowitz S. M., Nair N., Savage J., Simard C., Tian S., Waltz D., Xuan F., Rowe S. M., Jain R., Magazzu G.   +21 more
openaire   +3 more sources

Lung function improvement on triple modulators: high-resolution, nationwide data from the Danish Cystic Fibrosis Cohort

ERJ Open Research
Background People living with cystic fibrosis in Denmark had early, universal access to triple modulator treatment with elexacaftor/tezacaftor/ivacaftor.
Christian Leo-Hansen, Daniel Faurholt-Jepsen, Tavs Qvist, Christine Højte, Bibi U. Nielsen, Thomas Bryrup, Esben H. Henriksen, Terese Katzenstein, Marianne Skov, Inger H.M. Mathiesen, Majbritt Jeppesen, Søren Jensen-Fangel, Hanne V. Olesen, Frederik Fouirnaies Buchvald, Kim Gjerum Nielsen, Espen Jimenez-Solem, Christian Ritz, Tacjana Pressler, Mette F. Olsen, the TransformCF Study Group   +19 more
doaj   +1 more source

Determining the Minimal Clinically Important Difference of the 40‐Item Smell Identification Test in People With Cystic Fibrosis

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are highly prevalent among people with cystic fibrosis (PwCF) and negatively impact quality of life. The 40‐item Smell Identification Test (SIT) is widely used to assess psychophysical olfaction, but a CF‐specific minimal clinically important difference (MCID) has not been ...
Eugene Oh, Jessa E. Miller, Michelle J. Lee, Anna Zemke, David Baraghoshi, Matthew J. Strand, Jeremiah A. Alt, Todd E. Bodner, Michael Chang, Naweed I. Chowdhury, Patricia H. Eshaghian, Anne E. Getz, Jennifer L. Goralski, David A. Gudis, Peter H. Hwang, Ashoke Khanwalkar, Adam J. Kimple, Jivianne T. Lee, Douglas A. Li, Jess C. Mace, Jayakar V. Nayak, Jonathan B. Overdevest, Zara Patel, Daniella K. Safatian, Rodney J. Schlosser, Brent Senior, Amanda L. Stapleton, Timothy L. Smith, Zachary M. Soler, Jeffrey D. Suh, Grant A. Turner, Marilene B. Wang, Milene T. Saavedra, Jennifer L. Taylor‐Cousar, Daniel M. Beswick   +34 more
wiley   +1 more source

Pregnancy‐related effect on elexacaftor, tezacaftor and ivacaftor pharmacokinetics in women with cystic fibrosis

British Journal of Clinical Pharmacology, EarlyView.
Aim The number of pregnancies among women with cystic fibrosis (wwCF) has steadily increased over the past decade. However, the pharmacokinetics (PK) of elexacaftor–tezacaftor–ivacaftor (ETI) during gestation remains uncharacterized, despite its widespread use in this population.
Paulette Magnas, Naim Bouazza, Frantz Foissac, Saïk Urien, Léo Froelicher‐Bournaud, Gabrielle Lui, Victoria Guillemart, Nicolas Carlier, Jennifer Da Silva, Johanna Fesenbeckh, Reem Kanaan, Isabelle Honoré, Clémence Martin, Jean‐Marc Treluyer, Vassilis Tsatsaris, Pierre‐Régis Burgel, Sihem Benaboud   +16 more
wiley   +1 more source

Impact of cystic fibrosis transmembrane conductance regulator modulator therapies on liver stiffness and liver enzymes: An observational perspective single‐center cohort study

JPGN Reports, EarlyView.
Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano, Elisabetta Augustoni, Antonio Pizzol, Anna Opramolla, Cristina Chiadò, Anna Zagaria, Irene Esposito, Giulia Roberto, Alice Ponte, Andrea Evangelista, Paola Quarello, Michele Pinon, Pier Luigi Calvo   +12 more
wiley   +1 more source

Elexacaftor/tezacaftor/ivacaftor corrects monocyte microbicidal deficiency in cystic fibrosis

European Respiratory Journal, 2022
BackgroundCystic fibrosis (CF), which is caused by mutations in the CF transmembrane conductance regulator (CFTR), is characterised by chronic bacterial lung infection and inflammation. In CF, monocytes and monocyte-derived macrophages have been shown to display defective phagocytosis and antimicrobial activity against relevant lung pathogens ...
Luca Cavinato, Francesco R. Luly, Valentina Pastore, Daniele Chiappetta, Gloria Sangiorgi, Eva Ferrara, Pia Baiocchi, Giuseppe Mandarello, Giuseppe Cimino, Paola Del Porto, Fiorentina Ascenzioni   +10 more
openaire   +2 more sources

Mejoría de la inflamación intestinal tras tratamiento con moduladores de la proteína CFTR en pacientes con fibrosis quística

Anales de Pediatría
Resumen: Introducción: Los tratamientos con moduladores de la proteína CFTR han mejorado la salud respiratoria y digestiva de los pacientes con fibrosis quística.
Ruth García Romero, Concepción López Cárdenes, Elena Crehuá Gaudiza, Marina Álvarez Beltrán, Mercedes Murray Hurtado, Carlos Tutau Gómez, Inés Loverdos Eseverri, Encarni Torcuato Rubio, Camila García Volpe, Enrique Salcedo Lobato, María Medina Martínez, Carmen Martin Fernández, Ana Moreno Álvarez, Ana Reyes Domínguez, David González Jiménez   +14 more
doaj   +1 more source