High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial - PubMed
3 months ago
- #nusinersen
- #spinal muscular atrophy
- #clinical trial
- High-dose nusinersen evaluated in phase 3 randomized trial for spinal muscular atrophy (SMA).
- DEVOTE trial assessed efficacy and safety of high-dose nusinersen (50-mg loading, 28-mg maintenance dose).
- Primary endpoint met: significant improvement in CHOP-INTEND scores for high-dose group vs. sham.
- Safety profile of high-dose nusinersen similar to standard 12/12 mg regimen.
- Study supports benefits of high-dose nusinersen for SMA with well-tolerated safety.
- Trial registered on ClinicalTrials.gov (NCT04089566) and EudraCT (2019-002663-10).
- Authors report various conflicts of interest, including funding and advisory roles with pharmaceutical companies.