مؤتمر
PROCEEDINGS OF THE XXI CONGRESS OF THE ITALIAN ASSOCIATION OF MYOLOGY: Digital EditionDecember 01-04, 2021 Neurofilament light chain and Profilin-1 in adult SMA patients under nusinersen treatment: 26-months follow-up
| العنوان: | PROCEEDINGS OF THE XXI CONGRESS OF THE ITALIAN ASSOCIATION OF MYOLOGY: Digital EditionDecember 01-04, 2021 Neurofilament light chain and Profilin-1 in adult SMA patients under nusinersen treatment: 26-months follow-up |
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| المؤلفون: | Giulia Musso, Virginia Bozzoni, Luca Caumo, Luca Bello, Chiara Cosma, Gianni Sorarù, Mario Plebani, Elena Pegoraro |
| المساهمون: | Musso, Giulia, Bozzoni, Virginia, Caumo, Luca, Bello, Luca, Cosma, Chiara, Soraru', Gianni, Plebani, Mario, Pegoraro, Elena |
| سنة النشر: | 2021 |
| المجموعة: | Padua Research Archive (IRIS - Università degli Studi di Padova) |
| الوصف: | As an update of our study in 30 adult SMA type 3 patients we tested as potential biomarkers Neurofilament light chain (NfL) and Profilin-1 (PFN-1) during a 26 months follow-up (timepoint M6). PFN-1 is a small actin-binding protein required in both the presynaptic and the postsynaptic compartment with a role in regulating cytoskeletal architecture and dynamics of neurons (Witke, 2004). CSF NfL was tested with an enzyme-linked immunosorbent assay (ELISA) kit (UmanDiagnostics, Sweden). PFN-1 was measured with a commercial manual ELISA kit (Cusabio, China) in CSF samples of 6 patients (3 sitters 3 walkers) at time points L1, L3 and M2 and in every available serum sample from time point L1 to time point M6; 19 serum samples of healthy donors as a control group. Mean NfL at M6 was 381.83 ± 455.71 ng/l, slightly higher than baseline but not significantly different from M3. PFN-1 concentration in all 18 CSF samples was below the lower limit of quantification (< 31.25 ng/l). Serum PFN-1 at baseline was higher in SMA group than in healthy controls (mean 1016 vs 608 ng/l, p = 0.001 Student’s t test). PFN-1 showed a complex dynamic during loading phase, with a significant reduction at L4 compared to baseline. No correlation was found with NfL and motor scores at each time point. PFN-1 as an exploratory cytoskeletal biomarker changed significantly during the first two months of treatment. To our knowledge this is the first report of PFN-1 determination in serum of SMA patients. |
| نوع الوثيقة: | conference object |
| وصف الملف: | ELETTRONICO |
| اللغة: | English |
| العلاقة: | info:eu-repo/semantics/altIdentifier/pmid/34993420; ispartofbook:Neurofilament light chain and Profilin-1 in adult SMA patients under nusinersen treatment: 26-months follow-up; XXI CONGRESS OF THE ITALIAN ASSOCIATION OF MYOLOGY; volume:40 (4 Suppl 1); firstpage:62; lastpage:63; numberofpages:2; journal:ACTA MYOLOGICA; http://hdl.handle.net/11577/3413244Test |
| DOI: | 10.36185/2532-1900-056 |
| الإتاحة: | https://doi.org/10.36185/2532-1900-056Test http://hdl.handle.net/11577/3413244Test |
| رقم الانضمام: | edsbas.D7D2A884 |
| قاعدة البيانات: | BASE |
| DOI: | 10.36185/2532-1900-056 |
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