Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.
about
Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice.The Effects of 2'-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials.Emerging therapies and challenges in spinal muscular atrophyDelivery is key: lessons learnt from developing splice-switching antisense therapiesLNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblastsISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.AMPA GluA1-flip targeted oligonucleotide therapy reduces neonatal seizures and hyperexcitabilityTowards improved oligonucleotide therapeutics through faster target binding kinetics.Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice.Gene therapy research in Asia.The clinical landscape for SMA in a new therapeutic era.Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy.Nusinersen: First Global Approval.Targeting Splicing in the Treatment of Human DiseaseThe ApoE receptors Vldlr and Apoer2 in central nervous system function and disease.The role of RNA alternative splicing in regulating cancer metabolism.Faulty RNA splicing: consequences and therapeutic opportunities in brain and muscle disorders.New treatments for serious conditions: ethical implications.How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophyCure SMA and our patient community celebrate the first approved drug for SMA.Therapeutic approaches for spinal muscular atrophy (SMA).Splice-Switching Therapy for Spinal Muscular Atrophy.Gene Therapy in the Nervous System: Failures and Successes.New insights into SMA pathogenesis: immune dysfunction and neuroinflammation.SMN Blood Levels in a Porcine Model of Spinal Muscular Atrophy.The effects of C5-substituted 2,4-diaminoquinazolines on selected transcript expression in spinal muscular atrophy cellsGapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy.Therapeutic strategies for spinal muscular atrophy: SMN and beyond.Neurodegenerative disease: models, mechanisms, and a new hope.Gene suppression approaches to neurodegeneration.Survival Motor Neuron Protein is Released from Cells in Exosomes: A Potential Biomarker for Spinal Muscular Atrophy.Dissociated excitation-contraction coupling in infantile Pompe disease.Therapies targeting DNA and RNA in Huntington's disease.Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing.Emerging Diagnostic and Therapeutic Strategies for Tauopathies.Treatment Advances in Spinal Muscular Atrophy.Natural history of infantile-onset spinal muscular atrophy.Motor milestone assessment of infants with spinal muscular atrophy using the hammersmith infant neurological Exam-Part 2: Experience from a nusinersen clinical study.Activation of a cryptic 5' splice site reverses the impact of pathogenic splice site mutations in the spinal muscular atrophy gene.Spinal Muscular Atrophy: The Treatment Approved.
P2860
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P2860
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.
description
2016 nî lūn-bûn
@nan
2016 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2016 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2016年の論文
@ja
2016年論文
@yue
2016年論文
@zh-hant
2016年論文
@zh-hk
2016年論文
@zh-mo
2016年論文
@zh-tw
2016年论文
@wuu
name
Treatment of infantile-onset s ...... -label, dose-escalation study.
@ast
Treatment of infantile-onset s ...... -label, dose-escalation study.
@en
type
label
Treatment of infantile-onset s ...... -label, dose-escalation study.
@ast
Treatment of infantile-onset s ...... -label, dose-escalation study.
@en
prefLabel
Treatment of infantile-onset s ...... -label, dose-escalation study.
@ast
Treatment of infantile-onset s ...... -label, dose-escalation study.
@en
P2093
P1433
P1476
Treatment of infantile-onset s ...... -label, dose-escalation study.
@en
P2093
C Frank Bennett
Claudia A Chiriboga
Daniel A Norris
Darryl C De Vivo
Eugene Schneider
Frank Rigo
Jacqueline Montes
Jiri Vajsar
John W Day
P304
P356
10.1016/S0140-6736(16)31408-8
P407
P577
2016-12-06T00:00:00Z