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Qualitative study exploring the views and perceptions of parents/carers of young children with CF regarding the introduction of CFTR modulator therapy (The REVEAL study; PaRents pErspectiVEs of KAftrio in chiLdren aged 2-5). [PDF]
BMJ Open Respir ResDavies SH +4 more
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Changes in vitamins and trace elements after initiation of highly effective CFTR modulator therapy in children and adults with cystic fibrosis - a real-life insight. [PDF]
Mol Cell PediatrFabricius D +6 more
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CFTR Modulator Therapy for Cystic Fibrosis
New England Journal of Medicine, 2017Cystic fibrosis is a disease of abnormal ion transport through epithelium that results in progressive lung disease as well as the involvement of other organs including the pancreas, gut, and liver. Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR), and inheritance is autosomal ...
Hartmut Grasemann
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Triple CFTR Modulator Therapy for Cystic Fibrosis
New England Journal of Medicine, 2018Cystic fibrosis is one of the most common life-threatening autosomal recessive disorders, affecting approximately 80,000 children and adults worldwide.1 It is caused by mutations that result in deficient or defective function of the cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel that is normally present in epithelial ...
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Unmasking hereditary hemochromatosis with CFTR modulator therapy
Journal of Clinical Images and Medical Case Reports, 2022Cystic Fibrosis (CF) is a genetic condition affecting the ability to excrete chloride, resulting in multi-system organ damage. Liver disease is one co-morbidity that affects 20-40% of people with CF. This case describes a 16-year-old male with CF (F508Del and Q493X mutations) who was started one lexacaftor/tezacaftor/ifacaftortriple CF Transmembrane ...
Richard Wong, DO +2 more
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CFTR Modulators: Current Status and Evolving Knowledge
Seminars in Respiratory and Critical Care Medicine, 2022AbstractIn the past decade, the medical management of people with cystic fibrosis (pwCF) has changed with the development of small molecules that partially restore the function of the defective CF transmembrane conductance regulator (CFTR) protein and are called CFTR modulators.
Lucile, Regard +3 more
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WNK1 and WNK4 modulate CFTR activity
Biochemical and Biophysical Research Communications, 2007The cystic fibrosis transmembrane conductance regulator (CFTR) is an ATP-gated chloride channel. WNK kinases are widely expressed modulators of ion transport. WNK1 and WNK4, two WNK kinases that are mutated in familial hyperkalemic hypertension (FHHt), are co-expressed with CFTR in several organs, raising the possibility that WNK kinases might alter ...
Chao-Ling, Yang +6 more
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CFTR Modulator Therapies for Cystic Fibrosis
Pediatric Allergy, Immunology, and Pulmonology, 2015The cloning of cystic fibrosis transmembrane conductance regulator (CFTR) set into motion a cascade of discoveries that have helped to reveal the underlying pathophysiologic basis of cystic fibrosis (CF). This discovery and the knowledge that followed have also provided the opportunity to target this basic defect, with the hope of reversing or ...
Aaron T, Trimble, Scott H, Donaldson
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The impact of CFTR modulators on bone health
Pediatric Pulmonology, 2022We are hopeful that CTFR modulation does have a positive impact of bone health, but we have concerns about this study design being used to support the hypothesis.
Elizabeth, Clarke +3 more
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