Risdiplam in SMA: feedback from adults in France

Practitioners at the Neuromuscular Reference Centre at the Henri-Mondor University Hospital (Créteil) report their real-life experience of treating six adults with SMA:

  • four with type II SMA and two with type III SMA participated in this observational study,
  • two had initially received intrathecal nusinersen and were then switched to risdiplam,
  • the observation period varied between 7 and 27 months, with motor function measurement (MFM32) serving as the primary endpoint,
  • treatment with risdiplam proved to be well tolerated and effective in achieving the expected functional gains, with particularly marked gains on dimension D3 of the MFM32.

Although this was only a small series of patients, the authors believe that risdiplam treatment is also effective in adults, with a significant lasting effect.

 

Risdiplam: therapeutic effects and tolerability in a small cohort of 6 adult type 2 and type 3 SMA patients. Severa G, Alfaro MDC, Alimi Ichola C, et al. Orphanet J Rare Dis. 2024.