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Page last checked: 14th December 2022
Last updated: 17th September 2021


This new clinical trial will evaluate whether treatment with a higher dose of nusinersen has the potential to improve clinical outcomes and address unmet medical needs in people with later-onset SMA who were previously treated with risdiplam. Up to 135 later-onset, non-ambulatory (unable to walk independently) people with SMA aged 5-39 years will be enrolled.

All participants must have been treated with the maximum dose of 5 mg of risdiplam before joining the study and be willing and able to change their treatment regimen to a higher dose of nusinersen. Further eligibility criteria also apply.

ClinicalTrials.Gov >