Demonstrating Bioequivalence for a Lumacaftor Monosubstance Formulation Versus Orkambi® (Lumacaftor/Ivacaftor) in Healthy Subjects [PDF]
Drugs in R&DBackground and Objective Lumacaftor is an active ingredient in the US Food and Drug Administration-approved combination medication Orkambi®, which is used for treating cystic fibrosis.
Alexandra Papaelias +2 more
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Spectral Analysis on Cuba-Lumacaftor: Cubane as Benzene Bioisosteres of Lumacaftor
ACS Omega, 2023 In this paper, we theoretically investigate the electronic structure and physical properties of cuba-lumacaftor, cubane as benzene bioisosteres of lumacaftor, stimulated by recent experimental reports [Wiesenfeldt M. P.; Nature2023, 618, 513-518]. The permanent electric dipole moments of cuba-lumacaftor in neutral, acidic, and alkaline environments are
Dongdong Wang +3 more
doaj +3 more sources
Mitochondrial Fragmentation Induced by the CFTR Modulators Lumacaftor and Ivacaftor in Immortalized Cystic Fibrosis Cell Lines [PDF]
CellsCystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene, which encodes a cAMP-activated chloride channel essential for epithelial function.
Camila Dib +8 more
doaj +2 more sources
Effect of CFTR modulators on glucose homeostasis in children and young adults with cystic fibrosis-related diabetes: a systematic review [PDF]
Frontiers in EndocrinologyIntroductionCystic 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 +6 more
doaj +2 more sources
Therapeutic Advances in Respiratory DiseaseBackground: A significant decline in pulmonary exacerbation rates has been reported in CF patients homozygous for F508del treated with lumacaftor/ivacaftor. However, it is still unclear whether this reduction reflects a diminished microbiological burden.
Lena Klingspor +2 more
exaly +2 more sources
Targeting programmed death ligand 1 for anticancer therapy using computational drug repurposing and molecular simulations [PDF]
Scientific ReportsDiscovering new drug candidates for complex diseases like cancer is a significant challenge in modern drug discovery. Drug repurposing provides a cost-effective and time-efficient strategy to identify existing drugs for novel therapeutic targets.
Mohd Shahnawaz Khan +4 more
doaj +2 more sources
Identification of potential SARS-CoV-2 inhibitors among well-tolerated drugs using drug repurposing and in vitro approaches [PDF]
Scientific ReportsThe 3C-like protease (3CLpro) is essential in the SARS-CoV-2 life cycle and a promising target for antiviral drug discovery, as no similar proteases exist in humans.
Betül Oruçoğlu +8 more
doaj +2 more sources
The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis [PDF]
Expert Opinion on Drug Safety, 2017 Lumacaftor-ivacaftor is indicated for treatment of cystic fibrosis (CF) in patients homozygous for the Phe-508del cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations. In clinical trials, treated patients showed improved pulmonary function, reduced pulmonary exacerbations, and other benefits.
Susanna Mccolley
exaly +3 more sources
Clinical Case Reports (discontinued), 2019 Key Clinical Message We report the initiation of CFTR modulator lumacaftor/ivacaftor combination (LUM/IVA) in two adolescents with cystic fibrosis who were treated with antifungal azoles (AZO) and tacrolimus (TCS) for liver transplantation.
Philippe Reix
exaly +2 more sources
Nutritional impact of CFTR modulators in children with cystic fibrosis
Frontiers in Pediatrics, 2023 BackgroundNutritional status is a major prognostic factor for breathing and the survival of patients with cystic fibrosis (CF). Since 2012, the development of CFTR modulators has considerably transformed the outcome of this disease.
Margaux Gaschignard +18 more
doaj +1 more source