Systemic administration of PRO051 in Duchenne's muscular dystrophy. - sprookjes - yvandenbroek - TriplyDB

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

http://www.wikidata.org/entity/Q34172661

about

Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives 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 Current and emerging treatment strategies for Duchenne muscular dystrophy Clinical utility of serum biomarkers in Duchenne muscular dystrophy Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis Duchenne Muscular Dystrophy: From Diagnosis to Therapy Dystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophies Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery Old and new therapeutic developments in steroid treatment in Duchenne muscular dystrophy A chemical view of oligonucleotides for exon skipping and related drug applications Non-coding RNAs in muscle dystrophies Antisense oligonucleotides: treating neurodegeneration at the level of RNA Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Potential molecular targeting of splice variants for cancer treatment Splicing therapy for neuromuscular disease Upper extremity 3-dimensional reachable workspace analysis in dystrophinopathy using Kinect Nutraceuticals and Their Potential to Treat Duchenne Muscular Dystrophy: Separating the Credible from the Conjecture Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy Knocking down disease: a progress report on siRNA therapeutics Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice Inflammation converts human mesoangioblasts into targets of alloreactive immune responses: implications for allogeneic cell therapy of DMD Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational The mdx mouse model as a surrogate for Duchenne muscular dystrophy Translational regulation of utrophin by miRNAs Targeting several CAG expansion diseases by a single antisense oligonucleotide RNA therapeutics: RNAi and antisense mechanisms and clinical applications Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy Silencing disease genes in the laboratory and the clinic The Dystrophin Complex: Structure, Function, and Implications for Therapy Enhancement of Blood-Brain Barrier Permeability and Delivery of Antisense Oligonucleotides or Plasmid DNA to the Brain by the Combination of Bubble Liposomes and High-Intensity Focused Ultrasound.The alternative heart: impact of alternative splicing in heart disease.Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.Rapamycin nanoparticles target defective autophagy in muscular dystrophy to enhance both strength and cardiac function Parathyroid hormone and parathyroid hormone type-1 receptor accelerate myocyte differentiation.Comparison of dixon and T1-weighted MR methods to assess the degree of fat infiltration in duchenne muscular dystrophy patients.Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.

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P2860

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

http://www.wikidata.org/entity/Q34172661

description

2011 nî lūn-bûn

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2011 թուականի Մարտին հրատարակուած գիտական յօդուած

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2011 թվականի մարտին հրատարակված գիտական հոդված

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2011年の論文

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2011年論文

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2011年論文

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2011年論文

@zh-hk

2011年論文

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2011年論文

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2011年论文

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name

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

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Systemic administration of PRO051 in Duchenne's muscular dystrophy.

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Systemic administration of PRO051 in Duchenne's muscular dystrophy.

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type

label

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

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Systemic administration of PRO051 in Duchenne's muscular dystrophy.

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Systemic administration of PRO051 in Duchenne's muscular dystrophy.

@nl

prefLabel

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

@ast

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

@en

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

@nl

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The New England Journal of Medicine

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Systemic administration of PRO051 in Duchenne's muscular dystrophy.

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Anneke A Janson

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Brigitte E Burm

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Gerard J Platenburg

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Giles V Campion

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Gunnar Buyse

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J M Ad Sitsen

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Jan J Verschuuren

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Jessica A Sipkens

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Johanna T van den Akker

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Judith C van Deutekom

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1513-1522

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scholarly article

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10.1056/NEJMOA1011367

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16

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364

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Annemieke Aartsma-Rus

Gert-Jan van Ommen

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2011-03-23T00:00:00Z

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21428760

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