In this study, 151 adult 5q-SMA patients with a wide range of ages, disease duration and motor function deficits received nusinersen. Expectations for treatment with nusinersen varied widely based on disease severity and pattern of symptoms and impairments. The majority of patients experienced relief of symptoms and motor deficits that was rated as mild or moderate. Despite the rather low level of perceived functional improvement, treatment satisfaction was high and recommendation rates were excellent.

The FU Berlin, the WWU Münster and the Einstein Center Digital Future have researched our digital supply and research platform. The results are available in a publication for an international readership.