Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial. - Wikidata - wikimedia - TriplyDB

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

http://www.wikidata.org/entity/Q34132007

about

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis Current and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implications New and emerging targeted therapies for cystic fibrosis Targeted therapies to improve CFTR function in cystic fibrosis New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicals Therapeutic suppression of premature termination codons: mechanisms and clinical considerations (review)The Evolution of Cystic Fibrosis Care Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis Current and Emerging Therapies for the Treatment of Cystic Fibrosis or Mitigation of Its Symptoms Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators Paediatrics: messages from Munich Novel small molecules potentiate premature termination codon readthrough by aminoglycosides Use 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 nephrology PharmGKB summary: very important pharmacogene information for CFTR Ataluren: 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 Product Nonsense 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.

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Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

http://www.wikidata.org/entity/Q34132007

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2014 nî lūn-bûn

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2014 թուականի Մայիսին հրատարակուած գիտական յօդուած

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2014年の論文

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The Lancet. Respiratory medicine

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Anne Malfroot

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Arthur Branstrom

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Christiane Knoop

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Cystic Fibrosis Ataluren Study Group

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Daniel B Rosenbluth

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Eitan Kerem

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Ellen M Welch

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Ernst Rietschel

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Frank J Accurso

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Gary L Elfring

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Spirometric reference values from a sample of the general U.S. population

Standardisation of spirometry

PTC124 targets genetic disorders caused by nonsense mutations.

Firefly luciferase as a tool in molecular and cell biology.

Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group.

General considerations for lung function testing.

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model

Genotype-phenotype correlation for pulmonary function in cystic fibrosis.

Rescue of nonsense mutations by amlexanox in human cells.

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.

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cystic fibrosis

phase III clinical trial

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4154311

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