Pivotal trial: CHERISH2,3
Study: A phase 3, multicenter, double-blind, randomized (2:1), sham-controlled trial
Treatment duration: 15 months
Participants: 126 patients with later-onset SMA, aged 2 to 9 years at screening
Primary endpoint: Change in motor function as measured by the Hammersmith Functional Motor Scale—Expanded (HFMSE)
Safety: The most common side effects were fever (43%), headache (29%), vomiting (29%), and back pain (25%)