Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Status:
Not yet recruiting
Trial end date:
2026-06-15
Target enrollment:
Participant gender:
Summary
Risdiplam Exchange (RISE) is a study of spinal muscular atrophy (SMA) patients who crossover
to 36 months of open-label risdiplam monotherapy following a comparable period of nusinersen
treatment. The schedule of assessments (SOAs) carry over seamlessly for the cohort from
studies done while treated with nusinersen and continue to track the most informative
outcomes from that trial (e.g. nine hole peg test and grip strength), while adding the Box
and Block Test (BBT) as an additional measure of upper limb endurance and function.