Health Canada approves Zolgensma®, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA)
Share this article 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 2,3 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 1 Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene to halt disease progression 1 DORVAL, QC, Dec. 16, 2020 /CNW/ - Novartis Pharmaceuticals Canada Inc. is pleased ...
Source: newswire.ca