Posted by Michael Wonder on 16 Dec 2020
Health Canada approves Zolgensma, the one-time gene therapy for paediatric patients with spinal muscular atrophy
16 December 2020 - Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene to halt disease progression.
Novartis Pharmaceuticals Canada is pleased to announce that Health Canada has approved Zolgensma (onasemnogene abeparvovec) an adeno-associated virus vector-based gene therapy indicated for the treatment of paediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 3 or fewer copies of SMN2 gene; or infantile-onset SMA.
