SMA Newsroom

Apr 29, 2022

New risdiplam data shows long-term improvement in babies with Type 1 SMA

The latest data from the FIREFISH study of risdiplam was presented at the 14th European Paediatric Neurology Society (EPNS) Congress.

New three year data, including one year data from the label extension, show long-term improvement in survival and motor milestones in babies with Type 1 SMA, confiming both efficacy and safety:

  • 91% of infants treated with Evrysdi in the FIREFISH study were still alive at three years
  • Infants treated with Evrysdi maintained or continued to improve in measures of motor function,including their ability to swallow, sit without support, stand with support and walk while holding on. Without treatment, these children cannot sit without support
  • The study also showed overall continued reductions in serious adverse events and hospitalisations over time