A real-world analysis is reinforcing gene therapy durability in severe infant spinal muscular atrophy. Researchers drew on Italy’s national registry to assess two-year outcomes following a single infusion delivered in early life, reporting sustained gains in motor milestones across the cohort. Registry-based evidence complements controlled trials by capturing variability in patient baseline characteristics, access to follow-on supportive care, and longer follow-up in routine clinical practice. For SMA treatment strategies, durability beyond the first year remains a major differentiator among approaches competing for early treatment lines. The findings add to a growing body of evidence that early, single-administration gene therapy can produce sustained functional changes, which may influence payer decisions and sequencing against SMN2-targeting therapies.