Eteplirsen for the treatment of Duchenne muscular dystrophy. - Wikidata - awgldk - TriplyDB

Eteplirsen for the treatment of Duchenne muscular dystrophy.

Eteplirsen for the treatment of Duchenne muscular dystrophy.

http://www.wikidata.org/entity/Q45855674

about

Facioscapulohumeral dystrophy: the path to consensus on pathophysiology Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials Current and emerging treatment strategies for Duchenne muscular dystrophy Cardiomyopathy in becker muscular dystrophy: Overview Clinical utility of serum biomarkers in Duchenne muscular dystrophy Duchenne Muscular Dystrophy: From Diagnosis to Therapy Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery A chemical view of oligonucleotides for exon skipping and related drug applications Upper extremity 3-dimensional reachable workspace analysis in dystrophinopathy using Kinect Molecular Biology and Infection of Hepatitis E Virus Advances in gene therapy for muscular dystrophies Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy Non-invasive MRI and spectroscopy of mdx mice reveal temporal changes in dystrophic muscle imaging and in energy deficits Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy Upper Limb Evaluation in Duchenne Muscular Dystrophy: Fat-Water Quantification by MRI, Muscle Force and Function Define Endpoints for Clinical Trials Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study Delivery is key: lessons learnt from developing splice-switching antisense 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.What Can We Learn From Clinical Trials of Exon Skipping for DMD?Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx mice Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.MicroRNA-486-dependent modulation of DOCK3/PTEN/AKT signaling pathways improves muscular dystrophy-associated symptoms The notorious R.N.A. in the spotlight - drug or target for the treatment of disease Design and evaluation of locked nucleic acid-based splice-switching oligonucleotides in vitro.Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice Caspase-12 ablation preserves muscle function in the mdx mouse.A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.A comprehensive database of Duchenne and Becker muscular dystrophy patients (0-18 years old) in East China Reachable workspace in facioscapulohumeral muscular dystrophy (FSHD) by Kinect Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies.Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients.Dystrophin quantification: Biological and translational research implications.Gene therapy for muscular dystrophy: moving the field forward.High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy.Upper limb strength and function changes during a one-year follow-up in non-ambulant patients with Duchenne Muscular Dystrophy: an observational multicenter trial.

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P2860

Eteplirsen for the treatment of Duchenne muscular dystrophy.

http://www.wikidata.org/entity/Q45855674

description

2013 nî lūn-bûn

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2013年の論文

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2013年学术文章

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2013年学术文章

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2013年学术文章

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2013年学术文章

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2013年学术文章

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2013年学术文章

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2013年學術文章

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2013年學術文章

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name

Eteplirsen for the treatment of Duchenne muscular dystrophy.

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Eteplirsen for the treatment of Duchenne muscular dystrophy.

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type

label

Eteplirsen for the treatment of Duchenne muscular dystrophy.

@en

Eteplirsen for the treatment of Duchenne muscular dystrophy.

@nl

prefLabel

Eteplirsen for the treatment of Duchenne muscular dystrophy.

@en

Eteplirsen for the treatment of Duchenne muscular dystrophy.

@nl

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P1433

Annals of Neurology

P1476

Eteplirsen for the treatment of Duchenne muscular dystrophy.

@en

P2093

Ann Maria Gomez

http://www.w3.org/2001/XMLSchema#string

Chris Shilling

http://www.w3.org/2001/XMLSchema#string

Christopher M Walker

http://www.w3.org/2001/XMLSchema#string

Edward M Kaye

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Eteplirsen Study Group

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Hugh D Allen

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Janaiah Kota

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Jay B Saoud

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Jerry R Mendell

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John R Kean

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P2860

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

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

A randomized study of alglucosidase alfa in late-onset Pompe's disease.

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Dystrophin and mutations: one gene, several proteins, multiple phenotypes.

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.

Therapeutics in duchenne muscular dystrophy.

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.

P304

637-647

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P31

scholarly article

P356

10.1002/ANA.23982

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P433

5

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P478

74

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Kevin M. Flanigan

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2013-09-10T00:00:00Z

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262044004

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23907995

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