Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.
about
Potentiators (specific therapies for class III and IV mutations) for cystic fibrosisCurrent and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implicationsNew and emerging targeted therapies for cystic fibrosisTargeted therapies to improve CFTR function in cystic fibrosisNew approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicalsTherapeutic suppression of premature termination codons: mechanisms and clinical considerations (review)The Evolution of Cystic Fibrosis CareCystic 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 SymptomsRobust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric ModulatorsPaediatrics: messages from MunichNovel small molecules potentiate premature termination codon readthrough by aminoglycosidesUse of FEV1 in cystic fibrosis epidemiologic studies and clinical trials: A statistical perspective for the clinical researcher.Aminoglycoside-stimulated readthrough of premature termination codons in selected genes involved in primary ciliary dyskinesia.Aminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes.CFTR Modulators for the Treatment of Cystic Fibrosis.Translational read-through of the RP2 Arg120stop mutation in patient iPSC-derived retinal pigment epithelium cells.5-azacytidine inhibits nonsense-mediated decay in a MYC-dependent fashion.Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis.Translational nephrology: what translational research is and a bird's-eye view on translational research in nephrologyPharmGKB summary: very important pharmacogene information for CFTRAtaluren: first global approval.Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients.Recent advances in understanding and managing cystic fibrosis transmembrane conductance regulator dysfunction.From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations.Assessment of PAX6 alleles in 66 families with aniridia.Cystic Fibrosis Treatment: A Paradigm for New Pediatric Medicines, Globalization of Drug Development and the Role of the European Medicines Agency.Cystic fibrosis: a model system for precision medicine.CFTR: A New Horizon in the Pathomechanism and Treatment of Pancreatitis.Correctors and Potentiators Rescue Function of the Truncated W1282X-Cystic Fibrosis Transmembrane Regulator (CFTR) Translation ProductNonsense Suppression as an Approach to Treat Lysosomal Storage Diseases.Cystic fibrosis genetics: from molecular understanding to clinical application.Efficacy and adverse effects of drugs used to treat adult cystic fibrosis.Nonsense suppression therapies in ocular genetic diseases.A new era of personalized medicine for cystic fibrosis - at last!New and Emerging Treatments for Cystic Fibrosis.Repairing the basic defect in cystic fibrosis - one approach is not enough.
P2860
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P2860
Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.
description
2014 nî lūn-bûn
@nan
2014 թուականի Մայիսին հրատարակուած գիտական յօդուած
@hyw
2014 թվականի մայիսին հրատարակված գիտական հոդված
@hy
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
name
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@ast
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@en
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@nl
type
label
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@ast
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@en
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@nl
prefLabel
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@ast
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@en
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@nl
P2093
P2860
P50
P921
P1476
Ataluren for the treatment of ...... cebo-controlled phase 3 trial.
@en
P2093
Anne Malfroot
Arthur Branstrom
Christiane Knoop
Cystic Fibrosis Ataluren Study Group
Daniel B Rosenbluth
Eitan Kerem
Ellen M Welch
Ernst Rietschel
Frank J Accurso
Gary L Elfring
P2860
P304
P356
10.1016/S2213-2600(14)70100-6
P577
2014-05-15T00:00:00Z