Shots:

• The P-III STR1VE study involves assessing of Zolgensma in 22 patients with SMA Type1 + one or two copies of the SMN2 backup gene + bi-allelic SMN1 gene deletion or point mutations aged < 6mos. at the time of gene therapy
• The P-III STR1VE study results: alive patients (95%); patients reached 10.5mos. (86%); CHOP-INTEND score increases @1 & 3mos. after gene transfer (7 & 11.8 points);  8 patients could sit without support for 30sec. (median-age 12.5 mos.) with expected completion in 2020
• Zolgensma (onasemnogene abeparvovec-xioi- one-time infusion) is a gene therapy which introduces the copy of SMN gene using AAV9 and has received FDA’s BT designation & PR with an expected action date in May- 2019 and approval in Japan & EU in H2’19

Ref: Novartis | Image: Talkmarket