期刊
JOURNAL OF PEDIATRICS
卷 225, 期 -, 页码 252-+出版社
MOSBY-ELSEVIER
DOI: 10.1016/j.jpeds.2020.05.044
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资金
- AHRQ [K08 HS026510-01A1]
Spinal muscular atrophy is a neurodegenerative disease resulting from irreversible loss of anterior horn cells owing to biallelic deletions/mutations in the survival motor neuron (SMN) 1 gene. Gene replacement therapy using an adeno-associated virus vector containing the SMN gene was approved by the US Food and Drug Administration in May 2019. We report 2 cases of transient, drug-induced liver failure after this therapy.
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