Brazilian researchers have compiled data from the literature on one of three innovative drugs designed to treat children with type 1 spinal muscular atrophy (SMA1). The onasemnogene abeparvovec (Zolgensma®) is the first gene therapy product to have marketing authorisation for this indication:
- the authors selected four publications corresponding to three clinical trials (START, STR1VE-US and STR1VE-EU), none of which was comparative,
- with a minimum follow-up of 12 months, the survival rate of infants treated was 97.56%,
- the CHOP-INTEND score was greater than or equal to 40 in 87.28% of children,
- slightly more than half of them experienced treatment-related adverse events.
Although the efficacy of Zolgensma® has been well confirmed, the risk of complications associated with this new type of therapy and the lack of hindsight in these studies are matters for concern.
