Novartis to initiate SMART Phase 3b global study of Zolgensma in children up to 21 kg, building on real-world experience

  • SMART study to extend data beyond patient population studied in clinical trials
  • New clinical study to evaluate safety and efficacy of Zolgensma in children up to 21 kg, adding to real-world experience and regulatory approvals in Europe and Canada



Basel, April 23, 2021 - Novartis today announced plans to initiate SMART, a Phase 3b clinical study to evaluate the safety and efficacy of Zolgensma (onasemnogene abeparvovec) in young children with spinal muscular atrophy (SMA) weighing