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

• Main Authors: Rosanna Ruggiero (Author), Nunzia Balzano (Author), Maria Maddalena Nicoletti (Author), Gabriella di Mauro (Author), Federica Fraenza (Author), Maria Rosaria Campitiello (Author), Francesco Rossi (Author), Annalisa Capuano (Author)
• 格式: 圖書
• 出版: MDPI AG, 2024-03-01T00:00:00Z.
• 主題: article
• 在線閱讀: Connect to this object online.

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