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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| Autors principals: | , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
BMC
2025-05-01
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| Col·lecció: | Orphanet Journal of Rare Diseases |
| Matèries: | |
| Accés en línia: | https://doi.org/10.1186/s13023-025-03715-2 |
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