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ArticleNeurology and therapy2025

Early Intervention and Speed-to-Effect in Spinal Muscular Atrophy Type 1 Following Onasemnogene Abeparvovec Gene Replacement Therapy: Results of aPost-Hoc Analysis of Pooled Clinical Study Data.

Walter Toro et al.PubMed ↗Full text ↗Publisher ↗

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2025
2026
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Full record →Abstract, authors, funding and every citing paper · PMID 40622452