Results 41 to 50 of about 6,985 (222)
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
Modulator Therapy for Cystic Fibrosis: An Exploration of Current Research [PDF]
, 2020 Developing a drug therapy that addresses the root cause of cystic fibrosis (CF) by increasing CFTR protein levels has long been a research challenge. After genetic therapy failed because a suitable delivery system could not be found, researchers began ...
Rombocos, Jessalyn
core +1 more source
Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study [PDF]
, 2022 Rationale: The triple-combination regimen elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis and at least one F508del-CFTR allele in a phase 3, open-label, single ...
Ahluwalia, Neil +23 more
core +1 more source
Effect of CFTR Modulators on Respiratory Function in Adults with Cystic Fibrosis [PDF]
, 2021 Cystic Fibrosis (CF) is an autosomal recessive disorder that shortens one’s life due to its effect on the cystic fibrosis transmembrane regulator (CFTR) gene.
Severson, Melissa
core +2 more sources
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
Tezacaftor/Ivacaftor therapy has negligible effects on the cystic fibrosis gut microbiome
Microbiology Spectrum, 2023 People with cystic fibrosis (pwCF) experience a range of persistent gastrointestinal symptoms throughout life. There is evidence indicating interaction between the microbiota and gut pathophysiology in CF.
Ryan Marsh +7 more
doaj +1 more source
Overview of CFTR Modulators and Gene Therapy [PDF]
, 2020 Individuals with cystic fibrosis (CF) have seen a substantial change in their life expectancy since the introduction of coordinated multi-disciplinary care.
Kotsimbos, Tom +2 more
core +1 more source
Future Journal of Pharmaceutical Sciences, 2021 Background This study was designed to develop a reliable method for estimation of Ivacaftor and Tezacaftor in pure and its pharmaceutical dosage form by RP-HPLC in human plasma.
Madhuri Donakonda +4 more
doaj +1 more source
Frontiers in Pharmacology, 2023 The prevalence of mental health disorders is high among people with Cystic Fibrosis. The psychological symptoms in CF are associated with poor adherence, worse treatment outcomes, and greater health utilization/cost.
Hisham Ibrahim +19 more
doaj +1 more source