Postmarketing adverse events associated with onasemnogene abeparvovec: a real-world pharmacovigilance study

Postmarketing adverse events associated with onasemnogene abeparvovec: a real-world pharmacovigilance study

Abstract Background Onasemnogene abeparvovec (OA) is an adeno-associated virus vector-based gene therapy indicated for the treatment of paediatric patients with spinal muscular atrophy(SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene. This study focused on analysis of the pos...

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Bibliographic Details
Main Authors: Tianyu Chen, Qiying Chen, Jingfang Ye, Yuzhu Wu, Ting Liu, Yuezhen Zhang
Format: Article
Language:English
Published: BMC 2025-05-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Onasemnogene abeparvovec
OpenFDA
Adverse events
Pharmacovigilance
Online Access:https://doi.org/10.1186/s13023-025-03715-2
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https://doi.org/10.1186/s13023-025-03715-2

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