Treatment Advances in Spinal Muscular Atrophy

被引：0
作者：
Diana Bharucha-Goebel
Petra Kaufmann
机构：
[1] Children’s National Medical Center,Department of Neurology
[2] Neuromuscular and Neurogenetic Disorders of Childhoood Section (NNDCS)/NINDS/NIH,undefined
[3] National Center for Advancing Translational Sciences (NCATS)/NIH,undefined
来源：
Current Neurology and Neuroscience Reports | 2017年 / 17卷
关键词：
Spinal muscular atrophy; Clinical trials; Gene therapy; Natural history; Functional biomarkers; Newborn screening; Standard of care;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
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  • [1] Kolb SJ(2011)Spinal muscular atrophy: a timely review Arch Neurol 68 979-983
  • [2] Kissel JT(2017)Spinal muscular atrophy: factors that modulate motor neurone vulnerability Neurobiol Dis 102 11-20
  • [3] Wen-Yo T(2009)Spinal muscular atrophy : why do low levels of survival motor neuron protein make motor neurons sick? Nature Reviews 10 597-609
  • [4] Simpson JE(2015)Spinal muscular atrophy : diagnosis and management in a new therapeutic era Muscle Nerve 51 157-167
  • [5] Highley JR(2016)Type 0 spinal muscular atrophy: further delineation of prenatal and postnatal features in 16 patients J Neuromuscul Dis 3 487-495
  • [6] Heath PR(2017)Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy BMC Neurol 17 1-10
  • [7] Burghes AHM(2002)Outcome measures for pediatric spinal muscular atrophy Arch Neurol 59 1445-1450
  • [8] Beattie CE(2015)Upper limb evaluation and one-year follow up of non-ambulant patients with spinal muscular atrophy: an observational multicenter trial PLoS One 10 e021799-304
  • [9] Arnold WD(2017)Longitudinal characterization of biomarkers for spinal muscular atrophy Ann Clin Transl Neurol 4 292-1936
  • [10] Kassar D(2007)The changing natural history of spinal muscular atrophy type 1 Neurology 69 1931-817
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