Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
about
Current and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implicationsAnimal Models of Cystic Fibrosis Pathology: Phenotypic Parallels and DivergencesNew and emerging targeted therapies for cystic fibrosisManipulating proteostasis to repair the F508del-CFTR defect in cystic fibrosisYear in review 2015: Interstitial lung disease, pulmonary vascular disease, pulmonary function, sleep and ventilation, cystic fibrosis and paediatric lung diseaseAdvancing clinical development pathways for new CFTR modulators in cystic fibrosisValue of Organoids from Comparative Epithelia ModelsTargeted therapies to improve CFTR function in cystic fibrosisEpidemiology and genetics of cystic fibrosis in Asia: In preparation for the next-generation treatmentsNeuronal and Cardiovascular Potassium Channels as Therapeutic Drug Targets: Promise and PitfallsCystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectivesCFTR Modulators: Shedding Light on Precision Medicine for Cystic FibrosisCurrent and Emerging Therapies for the Treatment of Cystic Fibrosis or Mitigation of Its SymptomsRibosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional Expression DefectMechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic FibrosisIncreasing the Endoplasmic Reticulum Pool of the F508del Allele of the Cystic Fibrosis Transmembrane Conductance Regulator Leads to Greater Folding Correction by Small Molecule TherapeuticsBarriers to inhaled gene therapy of obstructive lung diseases: A reviewUse of FEV1 in cystic fibrosis epidemiologic studies and clinical trials: A statistical perspective for the clinical researcher.Background and EpidemiologyPediatric Pulmonology year in review 2015: Part 4.Cystic Fibrosis: Breakthrough Drugs at Break-the-Bank PricesAbdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings.Sweat test and cystic fibrosis: overview of test performance at public and private centers in the state of São Paulo, Brazil.Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia.Lumacaftor/ivacaftor combination for cystic fibrosis patients homozygous for Phe508del-CFTR.Lumacaftor and ivacaftor in the management of patients with cystic fibrosis: current evidence and future prospects.Exploring the genetic basis of early-onset chronic kidney disease.Simple image-based no-wash method for quantitative detection of surface expressed CFTR.Molecular modelling and molecular dynamics of CFTR.Evaporimeter and Bubble-Imaging Measures of Sweat Gland Secretion Rates.Cystic fibrosis lung environment and Pseudomonas aeruginosa infection.Improved fluorescence assays to measure the defects associated with F508del-CFTR allow identification of new active compounds.Hallmarks of therapeutic management of the cystic fibrosis functional landscape.Capturing the Direct Binding of CFTR Correctors to CFTR by Using Click Chemistry.Unravelling druggable signalling networks that control F508del-CFTR proteostasisFrom CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations.Modulating Innate and Adaptive Immunity by (R)-Roscovitine: Potential Therapeutic Opportunity in Cystic Fibrosis.Reducing Cardiovascular Risk Using Genomic Information in the Era of Precision Medicine.Precision Medicine: Clarity for the Complexity of Dementia.Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans
P2860
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P2860
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
2015年论文
@zh
2015年论文
@zh-cn
name
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
@ast
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
@en
type
label
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
@ast
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
@en
prefLabel
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
@ast
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
@en
P2093
P2860
P50
P356
P1476
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
@en
P2093
Anne Munck
Bonnie W Ramsey
Carla Colombo
David Waltz
Edward F McKone
Gautham Marigowda
Jane C Davies
Karen McCoy
Kris De Boeck
Marco Cipolli
P2860
P304
P356
10.1056/NEJMOA1409547
P407
P577
2015-05-17T00:00:00Z