Results 31 to 40 of about 2,457 (125)
Orphanet Journal of Rare Diseases, 2018 Background Lumacaftor/ivacaftor was approved by the Food and Drug Administration (FDA) as a combination treatment for Cystic Fibrosis (CF) patients who are homozygous for the F508del mutation.
Dolly Sharma +5 more
doaj +1 more source
Bridging for lung transplantation with lumacaftor/ivacaftor [PDF]
Breathe, 2018 The case of a young female cystic fibrosis patient, homozygous for delta F508 and with terminal respiratory insufficiency, who started treatment with lumacaftor/ivacaftor http://ow.ly/4I0t30kftDx.
Pedersen, Søren Sperling +3 more
openaire +4 more sources
Therapeutic Advances in Respiratory Disease, 2019 Background: Lumacaftor/ivacaftor combination therapy is efficacious and generally safe for patients with cystic fibrosis (CF) homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation.
Jaime L. Rubin +7 more
doaj +1 more source
Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype
Molecular Genetics & Genomic Medicine, 2021 Background New drugs that target the basic defect in cystic fibrosis (CF) patients may now be used in a large number of patients carrying responsive mutations. Nevertheless, further research is needed to extend the benefit of these treatments to patients
Vito Terlizzi +6 more
doaj +1 more source
Pediatric population with cystic fibrosis in the centre of Portugal: candidates for new therapies
Jornal de Pediatria, 2022 Objectives: Cystic fibrosis (CF) is a severe autosomal recessive disease that results from mutations in a gene encoding the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, a chloride channel.
Juliana Roda +6 more
doaj +1 more source
Predictive factors for lumacaftor/ivacaftor clinical response [PDF]
Journal of Cystic Fibrosis, 2019 Ivacaftor-lumacaftor combination therapy corrects the F508 del-CFTR mutated protein which causes Cystic Fibrosis. The clinical response of the patients treated with the combination therapy is highly variable. This study aimed to determine factors involved in the individual's response to lumacaftor-ivacaftor therapy.Sweat test was assessed at baseline ...
Masson, Alexandra +14 more
openaire +2 more sources
ERJ Open Research, 2020 Background Epithelial sodium channel (ENaC) is an important regulator of airway surface liquid volume; ENaC is hyperactivated in cystic fibrosis (CF). ENaC inhibition is a potential therapeutic target for CF.
Peter Nickolaus +4 more
doaj +1 more source
BMC Pharmacology and Toxicology, 2022 Background Lumacaftor/Ivacaftor (LUM-IVA), a cystic fibrosis transmembrane conductance regulator (CFTR) protein corrector-potentiator combination, improves lung function and reduces pulmonary exacerbations (PEx) in F508del homozygous CF patients. However,
Karin Yaacoby-Bianu +7 more
doaj +1 more source
Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy
Drug Design, Development and Therapy, 2019 GJ ConnettNational Institute for Health Research, Southampton Respiratory Biomedical Research Centre, University Hospital Southampton NHS Foundation Trust, Southampton, SO16 6YD, UKAbstract: Lumacaftor-ivacaftor is a combination of two small molecule ...
Connett GJ
doaj
EBioMedicineSummary: Background: In male mice, a circadian rhythm in myogenic reactivity influences the extent of brain injury following subarachnoid haemorrhage (SAH).
Danny D. Dinh +3 more
doaj +1 more source