SMA1 is a devastating monogenic neurodegenerative disease. Children with SMA1 are unable to sit unassisted, and by 6 months, almost none achieve a Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score ≥40 points (maximum 64). This study explored whether motor milestone achievement in SMA1 patients treated with a one-time dose of AVXS-101 gene replacement therapy was related to age at dosing. Twelve patients received an intravenous, proposed therapeutic dose of AVXS-101 (NCT02122952).

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