Reversing the irreversible: Another potential benefit of CFTR modulators
Sign Up to like & getrecommendations! Published in 2020 at "Pediatric Pulmonology"
DOI: 10.1002/ppul.25047
Abstract: To the Editor, Cystic fibrosis (CF) is a genetic condition resulting from a fundamental defect in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This defect leads to a reduction or absence of chloride transport… read more here.
Keywords: ivacaftor; lumacaftor ivacaftor; cftr; age ... See more keywords
Profiling the response to lumacaftor‐ivacaftor in children with cystic between fibrosis and new insight from a French‐Italian real‐life cohort
Sign Up to like & getrecommendations! Published in 2022 at "Pediatric Pulmonology"
DOI: 10.1002/ppul.26123
Abstract: Clinical trials for CFTR modulators consider mean changes of clinical status at the cohort level, and thus fail to assess the heterogeneity of the response. We aimed to study the different response profiles to lumacaftor‐ivacaftor… read more here.
Keywords: response; lumacaftor ivacaftor; cohort; children cystic ... See more keywords
Changes in Glucose Breath Test in Cystic Fibrosis Patients Treated with one Month of Lumacaftor/ivacaftor.
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DOI: 10.1097/mpg.0000000000003459
Abstract: BACKGROUND Alteration of the airway microbiota is a hallmark of cystic fibrosis (CF) pulmonary disease. Dysfunction of cystic fibrosis transmembrane regulator (CFTR) in the intestine also promotes changes in local microbiota such as small intestinal… read more here.
Keywords: cystic fibrosis; one month; luma iva; lumacaftor ivacaftor ... See more keywords
Effects of Lumacaftor-Ivacaftor on Airway Microbiota-Mycobiota and Inflammation in Patients with Cystic Fibrosis Appear To Be Linked to Pseudomonas aeruginosa Chronic Colonization
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DOI: 10.1128/spectrum.02251-22
Abstract: The management of cystic fibrosis has been transformed recently by the advent of CFTR modulators, including lumacaftor-ivacaftor. However, the effects of such therapies on the airway ecosystem, particularly on the microbiota-mycobiota and local inflammation, which… read more here.
Keywords: airway microbiota; inflammation; lumacaftor ivacaftor; microbiota mycobiota ... See more keywords
A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR
Sign Up to like & getrecommendations! Published in 2022 at "American Journal of Respiratory and Critical Care Medicine"
DOI: 10.1164/rccm.202204-0734oc
Abstract: Rationale Previous phase 3 trials showed that treatment with lumacaftor/ivacaftor was safe and efficacious in people aged ⩾2 years with cystic fibrosis (CF) homozygous for the F508del mutation in CFTR (CF transmembrane conductance regulator) (F/F… read more here.
Keywords: treatment; phase; lumacaftor ivacaftor; part ... See more keywords
Long-term effects of lumacaftor/ivacaftor on paranasal sinus abnormalities in children with cystic fibrosis detected with magnetic resonance imaging
Sign Up to like & getrecommendations! Published in 2023 at "Frontiers in Pharmacology"
DOI: 10.3389/fphar.2023.1161891
Abstract: Introduction: Chronic rhinosinusitis (CRS) usually presents with nasal congestion, rhinorrhea and anosmia impacts quality of life in cystic fibrosis (CF). Especially mucopyoceles pathognomonic for CRS in CF may cause complications such as spread of infection.… read more here.
Keywords: lumacaftor ivacaftor; age; mri; paranasal sinus ... See more keywords
Factors Predisposing the Response to Lumacaftor/Ivacaftor in People with Cystic Fibrosis
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DOI: 10.3390/jpm12020252
Abstract: Lumacaftor/ivacaftor (LUMA-IVA) therapy is prescribed to people with cystic fibrosis (pwCF) homozygous for the Phe508del-CFTR variant to restore CFTR protein function. There is, however, large inter-individual variability in treatment response. Here, we seek to identify… read more here.
Keywords: luma iva; people cystic; function; lumacaftor ivacaftor ... See more keywords