Biogen posts Phase 4 RESPOND results for nusinersen after onasemnogene abeparvovec
Results were posted for a completed Biogen trial testing Spinraza in children under 3 who had prior gene therapy for spinal muscular atrophy.
Biogen's single item of note this week is a results posting for a completed Phase 4 study.
- Nusinersen (Spinraza, BIIB058), Phase 4, spinal muscular atrophy: results were posted for the "RESPOND" study (NCT04488133), which was completed with an actual primary completion date of 2025-10-09 and results first posted 2026-10-30. The trial enrolled 46 participants and focused on children under the age of 3 who were previously treated with the gene therapy onasemnogene abeparvovec but were still facing health challenges related to their disease.1 The primary outcome was the Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score, measured up to Day 778.1
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