Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.
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Utility of Induced Pluripotent Stem Cells for the Study and Treatment of Genetic Diseases: Focus on Childhood Neurological DisordersIntegrated Safety Assessment of 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human VolunteersEmerging therapies and challenges in spinal muscular atrophyPharmacology, 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 therapiesEfficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide TreatmentGAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells.Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse ModelsLNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblastsBig win possible for Ionis/Biogen antisense drug in muscular atrophy.ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.A Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy ControlsNormalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration.AMPA GluA1-flip targeted oligonucleotide therapy reduces neonatal seizures and hyperexcitabilityRevised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool.Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy.Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience.Splice-switching antisense oligonucleotides as therapeutic drugsAltered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide TherapyGene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3.Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders.The clinical landscape for SMA in a new therapeutic era.Longitudinal characterization of biomarkers for spinal muscular atrophy.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.Population Pharmacokinetics of Nusinersen in the Cerebral Spinal Fluid and Plasma of Pediatric Patients With Spinal Muscular Atrophy Following Intrathecal Administrations.Non-coding RNAs as drug targets.Advances in modeling and treating spinal muscular atrophyTau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy.Nusinersen: First Global Approval.The chemical evolution of oligonucleotide therapies of clinical utility.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.Splice-Switching Therapy for Spinal Muscular Atrophy.Developing multidisciplinary clinics for neuromuscular care and research.Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases.Pharmacological therapeutics in Friedreich ataxia: the present state.
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P2860
Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.
description
2016 nî lūn-bûn
@nan
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
2016年论文
@zh
2016年论文
@zh-cn
name
Results from a phase 1 study o ...... with spinal muscular atrophy.
@ast
Results from a phase 1 study o ...... with spinal muscular atrophy.
@en
type
label
Results from a phase 1 study o ...... with spinal muscular atrophy.
@ast
Results from a phase 1 study o ...... with spinal muscular atrophy.
@en
prefLabel
Results from a phase 1 study o ...... with spinal muscular atrophy.
@ast
Results from a phase 1 study o ...... with spinal muscular atrophy.
@en
P2093
P2860
P1433
P1476
Results from a phase 1 study o ...... with spinal muscular atrophy.
@en
P2093
C Frank Bennett
Claudia A Chiriboga
Daniel A Norris
Darryl C De Vivo
Jacqueline Montes
Kathie M Bishop
Susan T Iannaccone
P2860
P304
P356
10.1212/WNL.0000000000002445
P407
P577
2016-02-10T00:00:00Z