自然史研究在脊髓性肌萎缩症中的应用(5)
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[27] Al-Zaidy SA，Kolb SJ，Lowes L，et al. AVXS-101 (Onasemnogene Abeparvovec) for SMA1：Comparative Study with a Prospective Natural History Cohort [J]. J Neuromuscul Dis，2019，6(3)：307-317.
[28] Kolb SJ，Coffey CS，Yankey JW，et al. Natural history of infantile-onset spinal muscular atrophy [J]. Ann Neurol，2017，82(6)，883-891.
[29] Chabanon A，Seferian AM，Daron A，et al. Prospective and longitudinal natural history study of patients with type 2 and 3 spinal muscular atrophy：baseline data NatHis-SMA study [J]. PLoS One，2018，13(7)：e0201004.
[30] Bertini E，Mercuri E. Motor neuron disease：A prospective natural history study of type 1 spinal muscular atrophy [J]. Nat Rev Neurol，2018，14(4)：197-198
(收稿日期：2020-02-11 本文編辑：封 华), 百拇医药(葛绣山 宋昉)
[25] Rudnik-Schoneborn S，Berg C，Zerres K，et al. Genotype-phenotype studies in infantile spinal muscular atrophy (SMA) type Ⅰ in Germany：implications for clinical trials and genetic counseling [J]. Clin Genet，2009，76(2)：168-178.
[26] Darbà J. Management and current status of spinal muscular atrophy：a retrospective multicentre claims database analysis [J]. Orphanet J Rare Dis，2020，15(1)：8.
[27] Al-Zaidy SA，Kolb SJ，Lowes L，et al. AVXS-101 (Onasemnogene Abeparvovec) for SMA1：Comparative Study with a Prospective Natural History Cohort [J]. J Neuromuscul Dis，2019，6(3)：307-317.
[28] Kolb SJ，Coffey CS，Yankey JW，et al. Natural history of infantile-onset spinal muscular atrophy [J]. Ann Neurol，2017，82(6)，883-891.
[29] Chabanon A，Seferian AM，Daron A，et al. Prospective and longitudinal natural history study of patients with type 2 and 3 spinal muscular atrophy：baseline data NatHis-SMA study [J]. PLoS One，2018，13(7)：e0201004.
[30] Bertini E，Mercuri E. Motor neuron disease：A prospective natural history study of type 1 spinal muscular atrophy [J]. Nat Rev Neurol，2018，14(4)：197-198
(收稿日期：2020-02-11 本文編辑：封 华), 百拇医药(葛绣山 宋昉)