Shots The Health Canada has approved Zolgensma (onasemnogene abeparvovec) for pediatric patients with 5q SMA with bi-allelic mutations in the SMN1 gene and 3 or fewer copies of SMN2 gene […]readmore
Tags : Zolgensma
Shots: The P-III STR1VE-EU study involves assessing Zolgensma (IV) in patients with SMA Type 1 aged <6mos. at the time of gene therapy, with 1 or 2 copies of the […]readmore
Shots: The EC’s approval is based on P-III STR1VE-US and P-I START studies assessing the efficacy & safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 […]readmore
Regulatory
Novartis’ Zolgensma (onasemnogene abeparvovec) Receives MHLW’s Approval for Spinal Muscular
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 […]readmore
Regulatory
Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) Receives FDA’s Approval for Spinal Muscular
Shots: The approval is based on ongoing P-III STR1VE & completed P-I START study result assessing Zolgensma (IV) in 21 & 15 patients with SMA and bi-allelic mutation in the […]readmore
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 […]readmore