Results 71 to 80 of about 2,379 (179)

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

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

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

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

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

The Changing Landscape of Treatment for Cystic Fibrosis Related Diabetes

Journal of Clinical & Translational Endocrinology
Objective: Patients with Cystic Fibrosis related diabetes [CFRD] are treated with insulin and high calorie diets to maintain body mass. The combined CFTR modulator elexacaftor/tezacaftor/ivacaftor [ETI] decreases pulmonary exacerbations and improves ...
Mehdia Amini, Kevin Yu, Jessica Liebich, Vaishaliben Ahir, Emily Wood, Stewart Albert, Sandeep Dhindsa   +6 more
doaj   +1 more source

Cystic Fibrosis: New Trends in Therapy Methods

Архивъ внутренней медицины
This review provides information on recent advancements in the treatment of cystic fi brosis and presents interim results from ongoing clinical trials. Various scientifi c databases, including Scopus, Web of Science, and EMBASE, were utilized during the ...
P. A. Suchkova, S. A. Panova, O. Ya. Lisenko, K. P. Raevskij   +3 more
doaj   +1 more source

Drug Responsiveness in Patient-Derived Rectal Organoids Correlates with Clinical Response in CF Subjects: A Real-Life Analysis

Scientia Pharmaceutica
Pharmacological modulators of CFTR have significantly changed the cystic fibrosis (CF) phenotype of subjects affected by this multi-organ disease.
Karina Kleinfelder, Paola Lecca, Roberta Valeria Latorre, Chiara Mortali, Sara Casati, Sofia Vanerio, Claudio Sorio, Paola Melotti   +7 more
doaj   +1 more source

Adult Diagnosis of Cystic Fibrosis: A Cause of Recurrent Pneumonia

Annals of Internal Medicine: Clinical Cases
We describe a patient presenting for evaluation of nearly annual pneumonias since the age of 9 years, eventually requiring multiple hospitalizations. Genetic testing revealed compound heterozygous mutations for 2184insA and L206W, indicative of cystic ...
Amira Elsabagh, Ashley Dean, Anthony Loschner   +2 more
doaj   +1 more source

Large variations in total and allele-specific transcript expression in a disease mutation-independent manner

Scientific Reports
Individuals with monogenic diseases, even those with identical disease-causing mutations, exhibit considerable clinical heterogeneity in severity and outcomes.
Moritz Freyberg, Merete Bewig, Giovana Bavia Bampi, Candela Manfredi, Disha Joshi, Robert Rauscher, Jeong S. Hong, Jörg Große-Onnebrink, Sivagurunathan Sutharsan, Florian Stehling, Ingrid Bobis, Manfred Ballmann, Eric J. Sorscher, Zoya Ignatova   +13 more
doaj   +1 more source