Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System

Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma<sup>®</sup>) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System

The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma<sup>®</sup>), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape. Although Zolgensma<sup>®</sup> therapy has proven to lead to functional improvements in SMA childre...

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Bibliographic Details
Main Authors: Rosanna Ruggiero, Nunzia Balzano, Maria Maddalena Nicoletti, Gabriella di Mauro, Federica Fraenza, Maria Rosaria Campitiello, Francesco Rossi, Annalisa Capuano
Format: Article
Language:English
Published: MDPI AG 2024-03-01
Series:Pharmaceuticals
Subjects:
gene therapy
orphan drug
safety data
pharmacovigilance
spinal muscular atrophy
Online Access:https://www.mdpi.com/1424-8247/17/3/394

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