nusinersen and Chemical-and-Drug-Induced-Liver-Injury

nusinersen has been researched along with Chemical-and-Drug-Induced-Liver-Injury* in 1 studies

Other Studies

1 other study(ies) available for nusinersen and Chemical-and-Drug-Induced-Liver-Injury

ArticleYear
Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.
    The Journal of pediatrics, 2020, Volume: 225

    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