Results 71 to 80 of about 2,330 (177)

Patient‐Derived Intestinal Organoids in the Global Cystic Fibrosis Landscape

Pediatric Pulmonology, Volume 61, Issue 4, April 2026.
ABSTRACT Cystic fibrosis (CF) care has advanced rapidly, yet diagnosis and inclusion in patient registries remain severely limited in low‐ and middle‐income countries (LMICs). Barriers include restricted newborn screening, limited availability of sweat chloride testing, and underrepresentation of non‐European CFTR variants in standard panels.
Suzanne Kroes, Jennifer L. Taylor‐Cousar, Marco Zampoli, Bülent Karadag, Cornelis K. van der Ent, Karin M. de Winter – de Groot, Jeffrey M. Beekman, Sacha Spelier   +7 more
wiley   +1 more source

Papilledema and hypervitaminosis A after elexacaftor/tezacaftor/ivacaftor for cystic fibrosis [PDF]

Canadian Journal of Ophthalmology, 2022
Matthew J. Miller, Rod Foroozan
openaire   +2 more sources

The long-term effect of elexacaftor/tezacaftor/ivacaftor on cardiorespiratory fitness in adolescent patients with cystic fibrosis: a pilot observational study

BMC Pulmonary Medicine
Background Physical activity is a crucial demand on cystic fibrosis treatment management. The highest value of oxygen uptake (VO2peak) is an appropriate tool to evaluate the physical activity in these patients.
Nela Stastna, Lenka Hrabovska, Pavel Homolka, Lukas Homola, Michal Svoboda, Kristian Brat, Libor Fila   +6 more
doaj   +1 more source

Ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis: a systematic review and economic evaluation

Health Technology Assessment
Background Cystic fibrosis is a life-limiting genetic condition that affects over 9000 people in England. Cystic fibrosis is usually diagnosed through newborn screening and causes symptoms throughout the body, including the lungs and digestive system ...
Steven J Edwards, Benjamin G Farrar, Kate Ennis, Nicole Downes, Victoria Wakefield, Isaac Mackenzie, Archie Walters, Tracey Jhita   +7 more
doaj   +1 more source

Economic impact of elexacaftor/tezacaftor/ivacaftor on healthcare expenditure in Canada

Journal of Cystic Fibrosis
The introduction of elexacaftor/tezacaftor/ivacaftor (ETI) has led to improved outcomes and survival in patients living with cystic fibrosis (PwCF) although imposes a substantial economic burden. Despite the reduced healthcare utilization that follows ETI initiation, the economic impact on healthcare spending is not well understood.
Stephen E. Congly, Ranjani Somayaji, Michael D. Parkins, Christina S. Thornton   +3 more
openaire   +2 more sources

Advances in the treatment of cystic fibrosis: CFTR modulators

Anales de Pediatría (English Edition)
Cystic fibrosis is a severe genetic disease caused by variants in the CFTR gene. Although it is a multisystem disease, respiratory involvement is the main cause of morbidity and mortality.
Maria Dolores Pastor-Vivero, Jordi Costa i Colomer, Carlos Martín de Vicente, Saioa Vicente-Santamaria, Ruth Garcia Romero, David González Jiménez, Carmen Luna Paredes   +6 more
doaj   +1 more source

Avances en el tratamiento de la fibrosis quística: los moduladores de la CFTR

Anales de Pediatría
Resumen: La fibrosis quística es una enfermedad genética y grave causada por variantes en el gen CFTR. Aunque se trata de una enfermedad multisistémica, la afectación respiratoria es la que produce mayor morbimortalidad.
Maria Dolores Pastor-Vivero, Jordi Costa i Colomer, Carlos Martín de Vicente, Saioa Vicente-Santamaria, Ruth García Romero, David González Jiménez, Carmen Luna Paredes   +6 more
doaj   +1 more source

Elexacaftor/tezacaftor/ivacaftor corrects salt-wasting in cystic fibrosis

Journal of Cystic Fibrosis
People with cystic fibrosis (pwCF) are at increased risk for fluid and electrolyte imbalances due to continuous salt loss. Congruently, guidelines advocate increased salt intake. However, pwCF are also at increased risk for developing cardiovascular disease.
Peder Berg, Amalie Quist Rousing, Søren Jensen-Fangel, Sascha Bandulik, Richard Warth, Mads Vaarby Sørensen, Majbritt Jeppesen, Jens Leipziger   +7 more
openaire   +3 more sources

Personalised CFTR Modulator Treatment Initiation and Monitoring in CF‐Related Liver Disease: When Less Is More

Respirology Case Reports
Hepatotoxicity due to Elexacaftor/Tezacaftor/Ivacaftor (ETI) use has been well documented. There are no dose adjustments or increased‐frequency monitoring algorithms recommended for people who experience elevated transaminases without cirrhosis, only ...
Sona Vekaria, Grace Kavanagh, Siobhain Mulrennan   +2 more
doaj   +1 more source

Scedosporium infection as a late complication after start of elexacaftor/tezacaftor/ivacaftor: two case reports. [PDF]

Respir Med Case Rep
Highly effective modulator treatment (HEMT) has resulted in an improved prognosis for people with cystic fibrosis (pwCF). In this case report, we present two pwCF with clinical stabilization after start of HEMT who developed complicated fungal infections
van Mourik P, van der Hout EC, Luijk HD, Hofland RW, Bruns AHW, Heijerman HGM, Bronsveld I.   +6 more
europepmc   +2 more sources