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Health Canada approves Zolgensma®, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA)


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SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2
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Zolgensma (onasemnogene abeparvovec) is approved for the treatment of pediatric SMA patients with 3 or fewer copies of the SMN2 gene, or infantile-onset SMA
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Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene to halt disease progression
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DORVAL, QC, Dec. 16, 2020 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased to announce that Health Canada has approved Zolgensma
® (onasemnogene abeparvovec) an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 ( ....

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