Results 71 to 80 of about 4,690 (200)

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

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

Extent of foetal exposure to maternal elexacaftor/tezacaftor/ivacaftor during pregnancy

British Journal of Pharmacology
AbstractBackground and PurposeCystic fibrosis (CF) patients are living longer and healthier due to improved treatments, e.g. cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI), with treatment possibly occurring in pregnancy. The risk of ETI to foetuses remain unknown.
Danni Li, Martin Donnelley, David Parsons, Mark D. Habgood, Elena K. Schneider‐Futschik   +4 more
openaire   +3 more sources

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

Improved Clinical Outcomes With Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis and Advanced Lung Disease: Real‐World Evidence From an Italian Single‐Center Study

Pharmacology Research & Perspectives
The combination of Elexacaftor/Tezacaftor/Ivacaftor (ETI) has resulted in a significant improvement in lung function and global clinical parameters, which have not been previously achieved with other CFTR modulators.
N. Perrotta, L. A. Fiorito, Gianfranco Casini, Rossella Gentile, R. Vescovo, A. Piciocchi, R. Lobello, C. Cappelli, R. Poscia, Giuseppe Cimino   +9 more
semanticscholar   +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

Elexacaftor-tezacaftor-ivacaftor pharmacokinetics with concurrent tacrolimus administration after lung transplant. [PDF]

J Cyst Fibros
CFTR modulators in post-transplant people with cystic fibrosis (pwCF) are less frequently used due to uncertainty regarding effectiveness and interactions with immunosuppressive agents. Elexacaftor/tezacaftor/ivacaftor (ETI) is a triple combination cystic fibrosis (CF) therapeutic with benefits in multiple organ systems where complications can impact ...
Guimbellot JS, Chalamalla A, Baker E, Ryan KJ, Dowell A, Abouelenein S, Bartlett LE, Bergeron J, Turner G, Acosta EP, Ramos KJ.   +10 more
europepmc   +3 more sources

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