Two Years of Improved Neurological Function With Nusinersen in a 48-Year-Old Patient With Spinal Muscular Atrophy Type 3
| Autor: | Gleydiane De Oliveira, Devon I. Rubin, Angelica R. Gicalone, Jaimin S. Shah, Christina R. Owens, Elliot L. Dimberg, Bjorn Oskarsson |
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| Rok vydání: | 2020 |
| Předmět: |
Adult patients
business.industry Neurological function Oligonucleotides Spinal muscular atrophy Middle Aged Oligonucleotides Antisense Spinal Muscular Atrophies of Childhood 030204 cardiovascular system & hematology medicine.disease Loading dose 03 medical and health sciences Treatment Outcome 0302 clinical medicine Anesthesia Antisense oligonucleotides Spinal Muscular Atrophy Type 3 Humans Medicine Female Nusinersen Muscle Strength business 030217 neurology & neurosurgery |
| Zdroj: | The Neurologist. 25:141-143 |
| ISSN: | 2331-2637 |
| DOI: | 10.1097/nrl.0000000000000284 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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