New real-life results for Zolgensma in 99 children with type I SMA, including 21 aged over 2 years and 7 weighing over 13.5 kg, collected from the six UK centres authorised to administer the gene therapy product, show :
- good tolerance of the product and its efficacy on motor function in older children (up to 7 years) and heavier children (up to 20 kg),
- a correlation between the patient’s weight and the duration of corticosteroid treatment on the one hand, and hepatotoxicity on the other, two parameters that need to be carefully monitored before and after treatment.
