Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial

标题
Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial
作者
关键词
Metachromatic leukodystrophy, Lysosomal storage disease, Enzyme replacement therapy, Intrathecal, Arylsulfatase A
出版物
MOLECULAR GENETICS AND METABOLISM
Volume -, Issue -, Pages -
出版商
Elsevier BV
发表日期
2020-07-16
DOI
10.1016/j.ymgme.2020.07.002

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