Forecast horizon — calibration-scored at resolution.
By 2028, long-term follow-up will show durability of Zolgensma motor benefit plateau at ≥5 years post-infusion in early-treated cohorts.
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Evidence stream
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Registry data
Apr 18, 2026
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Source publication
Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
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