Onasemnogene Abeparvovec for Spinal Muscular Atrophy: The Costlier Drug Ever.
作者信息
Mahajan Rajiv
机构信息
Department of Pharmacology, Adesh Institute of Medical Sciences and Research, Bathinda, Punjab, India.
出版信息
Int J Appl Basic Med Res. 2019 Jul-Sep;9(3):127-128. doi: 10.4103/ijabmr.IJABMR_190_19.
Abstract
摘要
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Nusinersen: A Treatment for Spinal Muscular Atrophy.依库珠单抗：治疗脊髓性肌萎缩症的一种方法。
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Phenotype modifiers of spinal muscular atrophy: the number of SMN2 gene copies, deletion in the NAIP gene and probably gender influence the course of the disease.脊髓性肌萎缩症的表型修饰因子：SMN2基因拷贝数、NAIP基因缺失以及可能的性别会影响疾病进程。
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Consensus statement for standard of care in spinal muscular atrophy.脊髓性肌萎缩症护理标准共识声明。
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