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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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)
Format: Book
Published: MDPI AG, 2024-03-01T00:00:00Z.
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3rd Floor Main Library

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