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WebZolgensma® (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based prescription gene therapy used to treat paediatric patients with spinal muscular atrophy (SMA). Originally developed by AveXis, the drug became a part of Novartis ’ portfolio after it acquired AveXis in May 2024 and renamed Novartis Gene Therapies. WebWelcome to the leading edge of gene therapy education The mission of the GT Network is to provide healthcare professionals with high quality, scientific information about gene therapy Monogenic Diseases An overview of genetic abnormalities and current treatment strategies for monogenic diseases Safety of Gene Therapy how many minutes in .3 hours
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