Nusinersen (Spinaraza®), a treatment for SMA available since 2017 in Europe, has been shown to be effective through clinical trials which mainly involved children. Over time, “real life” feedback also shows functional improvements in adults taking nusinersen.
To provide new data on this treatment in this population, a Belgian team has conducted two studies:
- a prospective study on the safety and efficacy of nusinersen for 14 months in 16 adults with type III SMA (14 participants) and type IV (2 participants),
- their data were compared with the data of a retrospective study concerning the natural history of 48 adolescents and adults with type II, III and IV SMA.
The results showed for the first time a significant improvement in the grip strength and motor function in hands of the participants. A significant improvement in the Medical Research Council (MRC) total scores has also been reported. In contrast, the seven participants who were still able to walk did not experience significant improvement at 14 months on the 6-minute walk test. Other parameters evaluated in this study (gross motor function or upper limbs, respiratory function, etc.) also did not show any significant effects of nusinersen.