Results 31 to 40 of about 3,864 (165)
Impact of CFTR Modulators on Beta-Cell Function in Children and Young Adults with Cystic Fibrosis [PDF]
, 2022 Background: To date, no consistent data are available on the possible impact of CFTR modulators on glucose metabolism. The aim of this study was to test the hypothesis that treatment with CFTR modulators is associated with an improvement in the key ...
Bonadonna R. C. +14 more
core +1 more source
Aplasia Cutis Congenita in the Setting of Maternal Cystic Fibrosis
Annals of Internal Medicine: Clinical Cases, 2022 Aplasia cutis congenita is a rare scalp defect. An affected neonate was delivered after in utero exposure beginning in the midtrimester to elexacaftor/tezacaftor/ivacaftor for maternal cystic fibrosis management. Although aplasia cutis congenita has many
Govind Kallumkal, Robert Egerman
doaj +1 more source
Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis [PDF]
, 2023 Cystic fibrosis; Guidelines; Variant-specific therapyFibrosis quística; Pautas; Terapia variante específicaFibrosi quística; Pautes; Teràpia variant específicaCystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic ...
Castellani, Carlo +6 more
core +1 more source
Novel reaction to new cystic fibrosis medication Trikafta
Clinical Case Reports, 2021 We present a novel case of an urticaria multiforme‐type drug reaction to the new cystic fibrosis medication Trikafta (elexacaftor + tezacaftor + ivacaftor).
Julian Stashower +3 more
doaj +1 more source
Pharmaceuticals, 2022 The use of modulator drugs that target the Cystic Fibrosis transmembrane conductance regulator (CFTR) is the final frontier in the treatment of Cystic Fibrosis (CF), a genetic multiorgan disease.
Giuseppe Migliorisi +7 more
doaj +1 more source
Pharmaceuticals, 2023 Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly
Mafalda Bacalhau +5 more
doaj +1 more source
Qualitative and quantitative evaluation of computed tomography changes in adults with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: a retrospective observational study [PDF]
, 2023 Introduction: The availability of highly effective triple cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapy with elexacaftor-tezacaftor-ivacaftor (ETI) has improved pulmonary outcomes and quality of life of people ...
Dettmer, Sabine +8 more
core +1 more source
Journal of Clinical & Translational Endocrinology, 2023 Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin +3 more
doaj +1 more source
Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis
Pharmacogenomics and Personalized Medicine, 2022 Iram Haq,1,2 Maryam Almulhem,1 Simone Soars,1 David Poulton,2,3 Malcolm Brodlie1,2 1Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle upon Tyne, UK; 2Paediatric Respiratory Medicine, Great North ...
Haq I +4 more
doaj
Clinical Case Reports, 2021 Elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane regulator (CFTR) modulator. It is known to be efficacious in stable patients with severe pneumopathy, but there are few data concerning its effectiveness during acute ...
Donatello Salvatore +4 more
doaj +1 more source