Is cerebrospinal fluid amyloid‐β42 a promising biomarker of response to nusinersen in adult spinal muscular atrophy patients? Article Swipe
Alessandro Introna
,
Giammarco Milella
,
Eustachio D’Errico
,
Angela Fraddosio
,
Gaspare Scaglione
,
Maria Ucci
,
Maddalena Ruggieri
,
Isabella Laura Simone
·
YOU?
·
· 2021
· Open Access
·
· DOI: https://doi.org/10.1002/mus.27212
YOU?
·
· 2021
· Open Access
·
· DOI: https://doi.org/10.1002/mus.27212
Introduction Nusinersen was approved as the first treatment for all types of spinal muscular atrophy (SMA), including adults with SMA types 2 and 3. Robust biomarkers of treatment response in SMA adults are lacking. Our aim was to examine cerebrospinal fluid (CSF) amyloid‐β40 (Aβ40) and amyloid‐β42 (Aβ42) peptides as biomarkers of treatment response. Methods Eight patients with SMA types 2 and 3 were recruited consecutively in a single‐center study. CSF was sampled at baseline, after a loading dose, and after three maintenance doses. Levels of Aβ42 and Aβ40 were evaluated for each CSF sampling. Wilcoxon matched‐pairs signed‐rank test was used to detect longitudinal changes. Results CSF levels of Aβ42 increased from baseline to day 420 (95% confidence interval, P = .018), with a significant increase at days 180 and 420 compared with days 0 and 300, respectively (95% confidence interval, P = .012 and P = .018). Discussion The maintenance and promotion of wellness of residual motor neurons mediated by the restored level of SMN protein due to nusinersen could result in an increased level of amyloid peptides.
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Metadata
- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.1002/mus.27212
- OA Status
- hybrid
- Cited By
- 15
- References
- 22
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W3133810913
All OpenAlex metadata
Raw OpenAlex JSON
- OpenAlex ID
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https://openalex.org/W3133810913Canonical identifier for this work in OpenAlex
- DOI
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https://doi.org/10.1002/mus.27212Digital Object Identifier
- Title
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Is cerebrospinal fluid amyloid‐β42 a promising biomarker of response to nusinersen in adult spinal muscular atrophy patients?Work title
- Type
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articleOpenAlex work type
- Language
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enPrimary language
- Publication year
-
2021Year of publication
- Publication date
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2021-03-04Full publication date if available
- Authors
-
Alessandro Introna, Giammarco Milella, Eustachio D’Errico, Angela Fraddosio, Gaspare Scaglione, Maria Ucci, Maddalena Ruggieri, Isabella Laura SimoneList of authors in order
- Landing page
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https://doi.org/10.1002/mus.27212Publisher landing page
- Open access
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YesWhether a free full text is available
- OA status
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hybridOpen access status per OpenAlex
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https://doi.org/10.1002/mus.27212Direct OA link when available
- Concepts
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SMA*, Cerebrospinal fluid, Spinal muscular atrophy, Medicine, Confidence interval, Biomarker, Internal medicine, Atrophy, Pathology, Gastroenterology, Disease, Biology, Biochemistry, Mathematics, CombinatoricsTop concepts (fields/topics) attached by OpenAlex
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15Total citation count in OpenAlex
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2025: 2, 2024: 3, 2023: 4, 2022: 3, 2021: 3Per-year citation counts (last 5 years)
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10Other works algorithmically related by OpenAlex
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