Ataluren treatment of patients with nonsense mutation dystrophinopathy. - Wikidata - wikimedia - TriplyDB

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

http://www.wikidata.org/entity/Q34561960

about

Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials Current and emerging treatment strategies for Duchenne muscular dystrophy Targeted therapies to improve CFTR function in cystic fibrosis Duchenne Muscular Dystrophy: From Diagnosis to Therapy Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: current and emerging options Translational readthrough potential of natural termination codons in eucaryotes--The impact of RNA sequence Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy Novel small molecules potentiate premature termination codon readthrough by aminoglycosides Aminoglycoside-stimulated readthrough of premature termination codons in selected genes involved in primary ciliary dyskinesia.Upper Limb Evaluation in Duchenne Muscular Dystrophy: Fat-Water Quantification by MRI, Muscle Force and Function Define Endpoints for Clinical Trials Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials Dystrophic Cardiomyopathy-Potential Role of Calcium in Pathogenesis, Treatment and Novel Therapies A Novel NF-κB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients With Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.Aminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes.The golden retriever model of Duchenne muscular dystrophy.Evaluation of muscular changes by ultrasound Nakagami imaging in Duchenne muscular dystrophy.A comprehensive database of Duchenne and Becker muscular dystrophy patients (0-18 years old) in East China Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.Ataluren: first global approval.Comparison of mutation profiles in the Duchenne muscular dystrophy gene among populations: implications for potential molecular therapies.Improving clinical trial design for Duchenne muscular dystrophy.Meeting Report: New Directions in the Biology and Disease of Skeletal Muscle 2014 Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies.Timed Rise from Floor as a Predictor of Disease Progression in Duchenne Muscular Dystrophy: An Observational Study.Naproxcinod shows significant advantages over naproxen in the mdx model of Duchenne Muscular Dystrophy.Rapid whole genome sequencing and precision neonatology Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy.Assessment of PAX6 alleles in 66 families with aniridia.Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice.An Overview of Cardiac Management in Neuromuscular Disease.DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study.The experiences of patients with Duchenne muscular dystrophy in facing and learning about their clinical conditions Comparison of Experimental Protocols of Physical Exercise for mdx Mice and Duchenne Muscular Dystrophy Patients.Looking Forward to New Therapies: A Personal Perspective on the Translational Landscape for Muscular Dystrophies.Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.Circulating Biomarkers for Duchenne Muscular Dystrophy.Identification of serum protein biomarkers for utrophin based DMD therapy.Eteplirsen in the treatment of Duchenne muscular dystrophy

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P2860

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

http://www.wikidata.org/entity/Q34561960

description

2014 nî lūn-bûn

@nan

2014 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2014 թվականի հոտեմբերին հրատարակված գիտական հոդված

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2014年の論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年论文

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name

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

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Ataluren treatment of patients with nonsense mutation dystrophinopathy.

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Ataluren treatment of patients with nonsense mutation dystrophinopathy.

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type

label

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

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Ataluren treatment of patients with nonsense mutation dystrophinopathy.

@en

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

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prefLabel

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

@ast

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

@en

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

@nl

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Muscle and Nerve

P1476

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

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Allan M Glanzman

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Allen Reha

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Barry Russman

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Brenda Wong

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Craig Campbell

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Eduard Gappmaier

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Erik Henricson

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Eugenio Mercuri

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Gary L Elfring

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H Lee Sweeney

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P2860

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Treatment of a methylmalonyl-CoA mutase stopcodon mutation

24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy

Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.

PTC124 targets genetic disorders caused by nonsense mutations.

Postnatal manipulation of Pax6 dosage reverses congenital tissue malformation defects.

ATS statement: guidelines for the six-minute walk test.

Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences.

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

A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene.

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477-487

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10.1002/MUS.24332

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4

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50

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Kevin M. Flanigan

Craig M. McDonald

Richard T Abresch

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2014-10-01T00:00:00Z

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264633028

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25042182

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4241581

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