The kick-off event for the MANAGE-SMA study will take place in Berlin on March 23 and 24, 2026.
The MANAGE-SMA study is the focus of the event. Treatment experience, treatment satisfaction and motor function in adult patients with 5q-SMA will be investigated. The therapies nusinersen 12 mg, risdiplam and the higher-dose nusinersen therapy (50/28 mg) will be examined.
The MANAGE-SMA study is designed as a longitudinal, non-interventional, observational, non-randomized, multi-center study with a total of 110 participants. The aim is to better understand the effect of the higher-dose nusinersen treatment regimen under everyday conditions in clinical practice.
The contents and objectives of the study will be presented in more detail at the kick-off event. In particular, this involves the recording of:
- patient-reported outcomes (PROMs)
- Treatment experience and treatment satisfaction
- motor functions and manual motor skills
- Performance Fatigability
- electrophysiological parameters
- Neurofilament light chain (NfL)
We are looking forward to an interesting kick-off event and will provide information on the key topics and results in one of the next newsletters.
The MANAGE-SMA kick-off event organized by APST and Biogen is not open to the public.
With best regards,
Your team from APST