Two Years of Improved Neurological Function With Nusinersen in a 48-Year-Old Patient With Spinal Muscular Atrophy Type 3

Jaimin S. Shah, Devon I. Rubin, Elliot L. Dimberg, Christina R. Owens, Angelica R. Gicalone, Gleydiane De Oliveira, Björn E. Oskarsson

Research output: Contribution to journalArticlepeer-review

Abstract

INTRODUCTION: Nusinersen antisense oligonucleotide infusions have been shown to be effective in the treatment spinal muscular atrophy. The majority of the evidence has been collected in young type 1 and type 2 patients, and evidence of efficacy in adult patients is limited. CASE REPORT: A 48-year-old woman with spinal muscular atrophy type 3 who has received the loading dose and 8 maintenance infusions over an 8-month period. Grip and pinch strength, measured by hand-held dynamometry measured at baseline and in 6 to 12 months interval improved over a 24-month period. She also reported multiple other subjective improvements in function. CONCLUSIONS: This is the first published case of nusinersen in a middle-aged adult with spinal muscular atrophy. Sustained clinically meaningful improvement may be possible with nusinersen initiation in mid adulthood.

Original languageEnglish (US)
Pages (from-to)141-143
Number of pages3
JournalThe neurologist
Volume25
Issue number5
DOIs
StatePublished - Sep 1 2020

ASJC Scopus subject areas

  • Clinical Neurology

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