Shots

• The Health Canada has approved Zolgensma (onasemnogene abeparvovec) for pediatric patients with 5q SMA with bi-allelic mutations in the SMN1 gene and 3 or fewer copies of SMN2 gene or infantile-onset SMA
• The efficacy and safety data supporting the approval of Zolgensma are derived from completed and ongoing studies in patients with SMA and 2 copies of SMN2 gene and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene
• Zolgensma (IV) is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene

 ­ Ref: Newswire Canada | Image: Novartis