Results 61 to 70 of about 2,230 (120)

Urticaria multiforme-like eruption due to a novel agent elexacaftor/tezacaftor/ivacaftor in a pediatric patient with cystic fibrosis

JAAD Case Reports, 2021
Rebecca H. Goldberg, BS, Natalie H. Matthews, MD, MPhil, Alexandra C. Hristov, MD, Frank Wang, MD   +3 more
doaj   +1 more source

Corrigendum: Elexacaftor-Tezacaftor-Ivacaftor treatment reduces abdominal symptoms in cystic fibrosis-early results obtained with the CF-specific CFAbd-Score

Frontiers in Pharmacology, 2023
Jochen G. Mainz, Jochen G. Mainz, Carlos Zagoya, Louise Polte, Lutz Naehrlich, Lutz Naehrlich, Lenny Sasse, Olaf Eickmeier, Christina Smaczny, Anton Barucha, Lilith Bechinger, Franziska Duckstein, Ludwik Kurzidim, Ludwik Kurzidim, Patience Eschenhagen, Laura Caley, Daniel Peckham, Daniel Peckham, Carsten Schwarz   +18 more
doaj   +1 more source

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

Pharmacological rescue of the G85E CFTR variant by preclinical and approved modulators

Frontiers in Pharmacology
IntroductionCystic Fibrosis (CF) is a genetic disease due to loss-of-function mutations of the CFTR channel. F508del is the most frequent mutation (70% of alleles in Italy), while other mutations have much lower frequency.
Valeria Tomati, Valeria Capurro, Emanuela Pesce, Cristina Pastorino, Elvira Sondo, Mariateresa Lena, Mariateresa Lena, Anna Borrelli, Federico Cresta, Stefano Pantano, Francesca Collini, Pietro Ripani, Vito Terlizzi, Cristina Fevola, Stefano Costa, Maria Cristina Lucanto, Federico Zara, Federico Zara, Tiziano Bandiera, Renata Bocciardi, Renata Bocciardi, Carlo Castellani, Luis J. V. Galietta, Nicoletta Pedemonte   +23 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

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

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

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

Correction to: Improved quality of life in cystic fibrosis patients observed up to 36 months after starting Elexacaftor/Tezacaftor/Ivacaftor treatment. [PDF]

J Patient Rep Outcomes
Buniotto F, Tridello G, De Scolari A, Meneghelli I, Pintani E, Perobelli S, Cipolli M.   +6 more
europepmc   +1 more source

Long-term impact of elexacaftor/tezacaftor/ivacaftor on small and large airways in people with cystic fibrosis aged ≥6 years: 24-month real-world evidence from the German Cystic Fibrosis Registry. [PDF]

ERJ Open Res
Dillenhöfer S, Schütz K, Burkhart M, Ellemunter H, Kappler M, Sieber S, Naehrlich L, Brinkmann F, Sutharsan S.   +8 more
europepmc   +1 more source