Onasemnogene Abeparvovec Administration via Peripherally Inserted Central Catheter: A Case Report

Onasemnogene abeparvovec (OA) is the approved intravenous gene therapy for the treatment of spinal muscular atrophy (SMA). A functional copy of the human <i>SMN1</i> gene was inserted into the target motor neuron cells via a viral vector, AAV9. In clinical trials, OA was infused through a peripheral...

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Detalhes bibliográficos

Principais autores:	Inmaculada Pitarch Castellano, Eduardo López Briz, Eugenia Ibáñez Albert, Cristina Aguado Codina, Teresa Sevilla, José L. Poveda Andrés
Formato:	Artigo
Idioma:	Inglês
Publicado em:	MDPI AG 2024-05-01
coleção:	Children
Assuntos:	onasemnogene abeparvovec peripherally inserted central catheter adeno-associated gene therapy spinal muscular atrophy adverse events
Acesso em linha:	https://www.mdpi.com/2227-9067/11/5/590
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