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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Principais autores:	Tianyu Chen, Qiying Chen, Jingfang Ye, Yuzhu Wu, Ting Liu, Yuezhen Zhang
格式:	Artigo
語言:	Inglês
出版:	BMC 2025-05-01
叢編:	Orphanet Journal of Rare Diseases
主題:	Onasemnogene abeparvovec OpenFDA Adverse events Pharmacovigilance
在線閱讀:	https://doi.org/10.1186/s13023-025-03715-2
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