Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.
case_report · Level V
Where this comes from
- Record sourced from PubMed, PMID 32473148.
- Also identified by DOI 10.1016/j.jpeds.2020.05.044 and PMC identifier 10152980.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
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.
Medical subject headings
- Chemical and Drug Induced Liver Injury
- Genetic Therapy
- Oligonucleotides
- Spinal Muscular Atrophies of Childhood