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

• Main Authors: Inmaculada Pitarch Castellano (Author), Eduardo López Briz (Author), Eugenia Ibáñez Albert (Author), Cristina Aguado Codina (Author), Teresa Sevilla (Author), José L. Poveda Andrés (Author)
• Format: Book
• Published: MDPI AG, 2024-05-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...