Article

Twelve-Month Outcomes of Intrathecal Vesemnogene Lantuparvovec for Spinal Muscular Atrophy in Children Younger than 24 Months in Low- and Middle-Income Countries

2026-05-30

Abstract excerpt

<h4>Introduction</h4> Spinal muscular atrophy (SMA) is a monogenic neuromuscular disease caused by mutations in the survival motor neuron 1 ( SMN1 ) gene. Onasemnogene abeparvovec is a U.S. FDA-approved single-dose gene therapy for SMA. Both its intravenous formulation (Zolgensma, approximately USD 2.13 million per patient) and intrathecal formulation (Itvisma, around USD 2.59 million per patient) are prohibitiv...

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Literature Corpus work: 40571645-caec-5e40-a647-ce70d5b57cd3
DOI: 10.64898/2026.05.27.26354188

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