Systemic administration of PRO051 in Duchenne's muscular dystrophy.
about
Drug treatment of Duchenne muscular dystrophy: available evidence and perspectivesExon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation studyCurrent and emerging treatment strategies for Duchenne muscular dystrophyClinical utility of serum biomarkers in Duchenne muscular dystrophyTherapeutic Oligonucleotides Targeting Liver Disease: TTR AmyloidosisDuchenne Muscular Dystrophy: From Diagnosis to TherapyDystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophiesAntisense mediated splicing modulation for inherited metabolic diseases: challenges for deliveryOld and new therapeutic developments in steroid treatment in Duchenne muscular dystrophyA chemical view of oligonucleotides for exon skipping and related drug applicationsNon-coding RNAs in muscle dystrophiesAntisense oligonucleotides: treating neurodegeneration at the level of RNAMolecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Potential molecular targeting of splice variants for cancer treatmentSplicing therapy for neuromuscular diseaseUpper extremity 3-dimensional reachable workspace analysis in dystrophinopathy using KinectNutraceuticals and Their Potential to Treat Duchenne Muscular Dystrophy: Separating the Credible from the ConjectureCurrent understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophyKnocking down disease: a progress report on siRNA therapeuticsAnimal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyRescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in miceInflammation converts human mesoangioblasts into targets of alloreactive immune responses: implications for allogeneic cell therapy of DMDTargeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug developmentGuidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translationalThe mdx mouse model as a surrogate for Duchenne muscular dystrophyTranslational regulation of utrophin by miRNAsTargeting several CAG expansion diseases by a single antisense oligonucleotideRNA therapeutics: RNAi and antisense mechanisms and clinical applicationsEvaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular DystrophySilencing disease genes in the laboratory and the clinicThe Dystrophin Complex: Structure, Function, and Implications for TherapyEnhancement 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 skippingLow dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout miceThe 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 functionParathyroid 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.
P2860
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P2860
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
description
2011 nî lūn-bûn
@nan
2011 թուականի Մարտին հրատարակուած գիտական յօդուած
@hyw
2011 թվականի մարտին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
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
type
label
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
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
P2093
P356
P1476
Systemic administration of PRO051 in Duchenne's muscular dystrophy.
@en
P2093
Anneke A Janson
Brigitte E Burm
Gerard J Platenburg
Giles V Campion
Gunnar Buyse
J M Ad Sitsen
Jan J Verschuuren
Jessica A Sipkens
Johanna T van den Akker
Judith C van Deutekom
P304
P356
10.1056/NEJMOA1011367
P407
P577
2011-03-23T00:00:00Z