• Zolgensma^® (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or SMA patients with up to three copies of the SMN2 gene
   
• Zolgensma has demonstrated significant and clinically meaningful therapeutic benefit in pre-symptomatic and symptomatic SMA, including prolonged event-free survival and achievement of motor milestones unseen in natural history of the disease and to date, sustained for 5 years post-dosing  
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