标题
Nusinersen for the treatment of spinal muscular atrophy
作者
关键词
-
出版物
Expert Review of Neurotherapeutics
Volume 17, Issue 10, Pages 955-962
出版商
Informa UK Limited
发表日期
2017-09-08
DOI
10.1080/14737175.2017.1364159
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial
- (2017) Enrico Bertini et al. LANCET NEUROLOGY
- Revised upper limb module for spinal muscular atrophy: Development of a new module
- (2017) Elena S. Mazzone et al. MUSCLE & NERVE
- Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
- (2016) Richard S Finkel et al. LANCET
- Six-minute walk test is reliable and valid in spinal muscular atrophy
- (2016) Sally Dunaway Young et al. MUSCLE & NERVE
- Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy
- (2016) Claudia A. Chiriboga et al. NEUROLOGY
- Splice-switching antisense oligonucleotides as therapeutic drugs
- (2016) Mallory A. Havens et al. NUCLEIC ACIDS RESEARCH
- Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study
- (2016) Stephen J. Kolb et al. Annals of Clinical and Translational Neurology
- SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA
- (2015) Vicki L. McGovern et al. HUMAN MOLECULAR GENETICS
- Spectrum of Neuropathophysiology in Spinal Muscular Atrophy Type I
- (2015) Brian N. Harding et al. JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY
- Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
- (2015) Kathrin Meyer et al. MOLECULAR THERAPY
- Observational study of spinal muscular atrophy type I and implications for clinical trials
- (2014) R. S. Finkel et al. NEUROLOGY
- SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
- (2014) N. A. Naryshkin et al. SCIENCE
- SMN Is Required for Sensory-Motor Circuit Function in Drosophila
- (2012) Wendy L. Imlach et al. CELL
- Activation of fast skeletal muscle troponin as a potential therapeutic approach for treating neuromuscular diseases
- (2012) Alan J Russell et al. NATURE MEDICINE
- Prospective cohort study of spinal muscular atrophy types 2 and 3
- (2012) P. Kaufmann et al. NEUROLOGY
- Evaluation of SMN Protein, Transcript, and Copy Number in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study
- (2012) Thomas O. Crawford et al. PLoS One
- Observational Study of Spinal Muscular Atrophy Type 2 and 3
- (2011) Petra Kaufmann ARCHIVES OF NEUROLOGY
- Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72 400 specimens
- (2011) Elaine A Sugarman et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Validation of the Expanded Hammersmith Functional Motor Scale in Spinal Muscular Atrophy Type II and III
- (2011) Allan M. Glanzman et al. JOURNAL OF CHILD NEUROLOGY
- Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy
- (2011) Cathleen M. Lutz et al. JOURNAL OF CLINICAL INVESTIGATION
- Early Functional Impairment of Sensory-Motor Connectivity in a Mouse Model of Spinal Muscular Atrophy
- (2011) George Z. Mentis et al. NEURON
- Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
- (2011) M. A. Passini et al. Science Translational Medicine
- Validation of the Childrenʼs Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
- (2011) Allan M. Glanzman et al. Pediatric Physical Therapy
- A Positive Modifier of Spinal Muscular Atrophy in the SMN2 Gene
- (2009) Thomas W. Prior et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?
- (2009) Arthur H. M. Burghes et al. NATURE REVIEWS NEUROSCIENCE
- Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice
- (2008) Yimin Hua et al. AMERICAN JOURNAL OF HUMAN GENETICS
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started