All Zolgensma (onasemnogene abeparvovec) articles
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NewsNew data revealed for Novartis SMA gene therapy
Novartis has revealed new data for its one-time gene therapy for spinal muscular atrophy (SMA) in older children.
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ArticleYear in review: EPR’s top stories of 2023
EPR wraps up the year with a selection of top stories from 2023, highlighting key trends and topics such as environmental monitoring (EM), quality (QA/QC), manufacturing, regulation and clinical development.
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ArticleRealising milestones with gene therapy for SMA
Novartis Gene Therapies' Vice President for Clinical Development & Analytics shares what shaped the success of one of the few commercially-available gene therapies.
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ArticleRealising the potential of AAV gene therapies
In this article, Dr Rajiv Vaidya, Head of Manufacturing Science & Technology at Andelyn Biosciences, explores strategies for improving the scalability and cost‑effectiveness of AAV production while maintaining regulatory compliance.
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NewsNovartis releases long-term data for SMA gene therapy
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.