Shots:

• The approval is based on P-I START- START long-term follow-up- P-III STR1VE-US- P-III SPR1NT and P-I/II STRONG (intrathecal). START and STR1VE-US assess Zolgensma (one-time- IV infusion) in symptomatic SMA Type 1 patients aged <6mos. with one/two copies of the SMN2 backup gene- or two copies of the SMN2 backup gene respectively
• The P-III SPR1NT study assess Zolgensma in pre-symptomatic patients aged <6wks. at age of dosing- genetically defined by bi-allelic deletion of SMN1 with 2/3 copies of SMN2. The studies demonstrated clinically benefit in symptomatic and pre-symptomatic SMA- including prolonged event-free survival and achievement of motor milestones unseen in the natural history of the disease 
• Zolgensma is a one-time gene therapy targeting the genetic root cause of the disease by replacing the function of the missing/ nonworking SMN1 gene with its expected MHLW’s reimbursement by the end H1’20

Click here ­to­ read full press release/ article 

Ref: Novartis | Image: Novartis