AAMS Neurology & Neurosciences · Vol. 7 · Issue 3 · 2026-03-01
Antisense Oligonucleotide Therapy for Spinal Muscular Atrophy Type 2: Long-Term Results
Bergström E, Ono S, Nkemelu OS
Karolinska Institute, Stockholm, Sweden; Nagoya University, Nagoya, Japan; University of Ibadan, Ibadan, Nigeria
Abstract
Long-term follow-up study reporting 7-year motor function, respiratory outcomes, and survival data for 280 patients with SMA type 2 treated with intrathecal nusinersen.
Keywords: spinal muscular atrophy, antisense oligonucleotide, nusinersen, motor function, long-term
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