An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
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From animal models to human disease: a genetic approach for personalized medicine in ALSThe P42 peptide and Peptide-based therapies for Huntington's diseaseToward precision medicine in amyotrophic lateral sclerosisClinical Trial Designs in Amyotrophic Lateral Sclerosis: Does One Design Fit All?Converging mechanisms in ALS and FTD: disrupted RNA and protein homeostasisState of play in amyotrophic lateral sclerosis geneticsAntisense oligonucleotides: treating neurodegeneration at the level of RNAAmyotrophic Lateral Sclerosis: An update for 2013 Clinical Features, Pathophysiology, Management and Therapeutic TrialsDynamic dual-isotope molecular imaging elucidates principles for optimizing intrathecal drug delivery.TDP-43 Proteinopathy and ALS: Insights into Disease Mechanisms and Therapeutic Targets.Emerging mechanisms of molecular pathology in ALSRNA-targeted Therapeutics for ALSAllele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patientsIntegrated Safety Assessment of 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human VolunteersAdeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model.Safety Evaluation of CNS Administered Biologics-Study Design, Data Interpretation, and Translation to the Clinic.Pharmacology, pharmacokinetics, and metabolism of the DNA-decoy AYX1 for the prevention of acute and chronic post-surgical painDelivery is key: lessons learnt from developing splice-switching antisense therapiesEvaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse ModelsMotor neuron expression of the voltage-gated calcium channel cacophony restores locomotion defects in a Drosophila, TDP-43 loss of function model of ALS.Genetic modulation of soluble Aβ rescues cognitive and synaptic impairment in a mouse model of Alzheimer's diseaseInherited Paediatric Motor Neuron Disorders: Beyond Spinal Muscular Atrophy.Genetics and ALS: Cause for Optimism.C9orf72 amyotrophic lateral sclerosis and frontotemporal dementia: gain or loss of function?Genetic heterogeneity of amyotrophic lateral sclerosis: implications for clinical practice and research.Targets for future clinical trials in Huntington's disease: what's in the pipeline?Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington's disease patients.Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.The AMPA receptor antagonist perampanel robustly rescues amyotrophic lateral sclerosis (ALS) pathology in sporadic ALS model mice.Antisense therapy in neurologyAberrant RNA homeostasis in amyotrophic lateral sclerosis: potential for new therapeutic targets?Towards a therapy for Angelman syndrome by targeting a long non-coding RNA.Gene therapy for the nervous system: challenges and new strategies.Protective and Toxic Neuroinflammation in Amyotrophic Lateral Sclerosis.In vivo kinetic approach reveals slow SOD1 turnover in the CNS.Morpholino-mediated SOD1 reduction ameliorates an amyotrophic lateral sclerosis disease phenotype.Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.Use of biomarkers in ALS drug development and clinical trialsGain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAsIntrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience.
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An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
@pt
bilimsel makale
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scientific article published on 29 March 2013
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
An antisense oligonucleotide a ...... randomised, first-in-man study
@en
An antisense oligonucleotide a ...... andomised, first-in-man study.
@nl
type
label
An antisense oligonucleotide a ...... randomised, first-in-man study
@en
An antisense oligonucleotide a ...... andomised, first-in-man study.
@nl
prefLabel
An antisense oligonucleotide a ...... randomised, first-in-man study
@en
An antisense oligonucleotide a ...... andomised, first-in-man study.
@nl
P2093
P2860
P50
P1433
P1476
An antisense oligonucleotide a ...... randomised, first-in-man study
@en
P2093
Alan Pestronk
C Frank Bennett
Daniel A Norris
David Schoenfeld
Ericka Simpson
Georgios Manousakis
Jeffrey Rothstein
Kathie M Bishop
Katie Alexander
Katy Mahoney
P2860
P304
P356
10.1016/S1474-4422(13)70061-9
P577
2013-03-29T00:00:00Z