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
Full Text
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.
This article is published open access by AAMS Neurology & Neurosciences. The complete peer-reviewed text can be read on the journal page.
Author Citation Metrics
- Bergström E — Citations: 294 · h-index: 3 · i10-index: 3
- Ono S — Citations: 2297 · h-index: 23 · i10-index: 46 · ORCID 0000-0002-6963-0911
Metrics are sourced from public bibliometric records (OpenAlex) and each author's Google Scholar profile.