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.