| Literature DB >> 32473148 |
Amy G Feldman1, Julie A Parsons2, Cullen M Dutmer2, Aravindhan Veerapandiyan3, Einar Hafberg4, Nolan Maloney5, Cara L Mack2.
Abstract
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.Entities:
Mesh：
Substances：
Year: 2020 PMID： 32473148 DOI： 10.1016/j.jpeds.2020.05.044
Source DB: PubMed Journal: J Pediatr ISSN： 0022-3476 Impact factor: 4.406