Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam. First, we want to quantify improvement of both ambulant and non-ambulant patients under treatment. A secondary objective would also be to identify suitable accelerometric measurements that are sensitive to change but also well correlated to other clinical scores.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
95th centile of stride velocity
Timeframe: 39 months
50th centile of stride velocity
Timeframe: 39 months
95th centile of stride length
Timeframe: 39 months
50th centile of stride length
Timeframe: 39 months
Upper limb function evolution in patient with SMA undergoing treatment
Timeframe: 39 months