European approval is based on the completed phase 3 STR1VE-US and phase 1 START trials that tested the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 ...

Novartis (NVS) already markets an IV-administered version of the gene therapy called Zolgensma. The product, which is one of ...

SMA is a genetic disease that causes muscle weakness ... which could potentially save each of her boys' lives. The one-dose IV drug treatment for each baby is a staggering $2.1million.

The mutation that causes Spinal Muscular Atrophy (SMA) can be detected in a prenatal screen. But a parent's decision to test for this terrible disease is not as easy as you would think.

Among SMA patients aged 2 to below 18 years ... Liver toxicity is a known problem with Zolgensma IV, and it’s highlighted in an FDA boxed warning. In Steer, the rate of liver toxicity was ...