nusinersen has been researched along with Chemical-and-Drug-Induced-Liver-Injury* in 1 studies
1 other study(ies) available for nusinersen and Chemical-and-Drug-Induced-Liver-Injury
Article | Year |
---|---|
Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.
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. Topics: Chemical and Drug Induced Liver Injury; Female; Genetic Therapy; Glucocorticoids; Humans; Infant; Male; Oligonucleotides; Prednisolone; Spinal Muscular Atrophies of Childhood | 2020 |