Article

Early Life Safety Profiling of Gene Therapy for Spinal Muscular Atrophy: A Case Series Analysis

2024-11-08

Abstract excerpt

The present study examines the safety profile of intravenous onasemnogene abeparvovec gene therapy in a real world setting, both alone or in combination with intrathecal antisense oligonucleotide nusinersen therapy in two cohorts of patients with spinal muscular atrophy (SMA). The first cohort included 8 presymptomatic infants treated solely with onasemnogene abeparvovec, while the second cohort comprised 6 sympto...

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Literature Corpus work: 85d869a1-c00f-5e0c-a31a-96d2ab8c714f
DOI: 10.1101/2024.11.04.24316576

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