Results 81 to 90 of about 2,330 (177)

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

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

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

Effect of CFTR modulators on glucose homeostasis in children and young adults with cystic fibrosis-related diabetes: a systematic review

Frontiers in Endocrinology
IntroductionCystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CFTR gene, leading to impaired chloride transport, thickened mucus, and multiorgan dysfunction. Among its complications, cystic fibrosis-related diabetes (CFRD)
Paola Giordano, Giuseppina Leonetti, Vanja Granberg, Rosa Maria Pia Casolino, Giuseppe Lassandro, Maurizio Delvecchio, Giovanna Linguiti   +6 more
doaj   +1 more source

An acneiform eruption associated with elexacaftor/tezacaftor/ivacaftor treatment [PDF]

Dermatology Online Journal, 2022
Breneman, Alyssa, Soliman, Yssra S, Gallitano, Stephanie M   +2 more
openaire   +3 more sources

People with cystic fibrosis with high sputum neutrophil elastase on elexacaftor-tezacaftor-ivacaftor exhibit worse pulmonary function and pro-inflammatory airway milieu

Scientific Reports
Cystic fibrosis (CF) is a genetic condition affecting over 100,000 individuals worldwide. Lung disease is the main cause of mortality in CF, with chronic neutrophilic inflammation as a hallmark.
Alexandre Cammarata-Mouchtouris, Diego Moncada Giraldo, Genoah L. Collins, Maria M. Parrilla, Susan O. Kim, Sarah Mansour, Vincent D. Giacalone, Kirsten A. Cottrill, Rabindra Tirouvanziam, Joshua D. Chandler   +9 more
doaj   +1 more source

Elexacaftor/Tezacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients With Cystic Fibrosis

Clinical and Translational Science
Elexacaftor/tezacaftor/ivacaftor (ETI) significantly improves treatment outcomes for people with cystic fibrosis (pwCF) with at least one F508del allele. In 2023, the Food and Drug Administration approved ETI for children with CF aged 2–5 years. However,
Ngoc Hoa Truong, Sihem Benaboud, Naïm Bouazza, Mahassen Barboura, Emmanuelle Bardin, Michel Miralles, Gabrielle Lui, Léo Froelicher‐Bournaud, Steeve Rouillon, Tiphaine Bihouee, Stéphanie Bui, Philippe Reix, Marie‐Laure Dalphin, Muriel Laurans, Jeanne Languepin, Harriet Corvol, Françoise Troussier, Laurence Weiss, Rames Cinthia, Aurélie Tatopoulos, Eric Deneuville, Raphael Chiron, Nathalie Stremler, Cathy Llerena, Sophie Ramel, Caroline Perisson, Véronique Houdoin, Marie Mittaine, Jean‐Marc Treluyer, Isabelle Sermet‐Gaudelus, Frantz Foissac, MODUL‐CF study group   +31 more
doaj   +1 more source

INITIATION OF ELEXACAFTOR-TEZACAFTOR-IVACAFTOR LEADING TO LUNG TRANSPLANTATION [PDF]

Chest, 2020
Travis Homan, Stephanie Link, Christopher Barrios   +2 more
openaire   +1 more source