A transnational registry containing real-life data from patients with SMN1-related proximal spinal muscular atrophy (SMA) has been set up in Germany, Austria and German-speaking Switzerland:
- data from patients who had received onasemnogene abeparvovec gene therapy (Zolgensma®) were analysed,
- 343 patients with an average age of 14 months at the time of Zolgensma® administration were included in the study, which represents the largest cohort in Europe,
- of these, 79 were presymptomatic,
- clinical benefit was achieved in the vast majority of cases, with children who had been treated very early showing a better outcome.
This study provides a wealth of information about the practices and limitations of Zolgensma® in SMA.
