Comparison of Nusinersen Monotherapy Versus Combination Therapy With Nusinersen and Onasemnogene Abeparvovec in Spinal Muscular Atrophy Type 1 Patients With Two SMN2 Copies: A Multicenter Study From T
5 hours ago
- #nusinersen
- #spinal muscular atrophy
- #onasemnogene abeparvovec
- Comparison of nusinersen monotherapy versus combination therapy (nusinersen and onasemnogene abeparvovec) in SMA type 1 patients with two SMN2 copies.
- Retrospective multicenter study involving 82 patients (42 monotherapy, 40 combination therapy).
- No significant difference in motor milestones, CHOP-INTEND scores, or respiratory/nutritional status between groups.
- All deaths (n=8) occurred in the monotherapy group among patients treated after 3 months of age (p=0.005).
- Early treatment initiation correlated with better feeding outcomes (OR: 0.740, p=0.024).
- Pre-treatment respiratory and feeding status were predictive of outcomes.
- Combination therapy showed no clear advantage over monotherapy; early treatment was the main determinant of outcomes.