Cerebrospinal fluid proteomic profiling in nusinersen-treated patients with spinal muscular atrophy
The approved antisense oligonucleotide nusinersen demonstrated convincing clinical results in patients with 5q-linked spinal muscular atrophy (SMA) but predictive biomarkers are lacking to date.
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| Main Authors: | , , , , , , , , , , , , |
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
2020
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| In: |
Journal of neurochemistry
Year: 2020, Volume: 153, Issue: 5, Pages: 650-661 |
| ISSN: | 1471-4159 |
| DOI: | 10.1111/jnc.14953 |
| Online Access: | Verlag, kostenfrei, Volltext: https://doi.org/10.1111/jnc.14953 Verlag, kostenfrei, Volltext: https://onlinelibrary.wiley.com/doi/10.1111/jnc.14953 
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| Author Notes: | Tobias Kessler, Pauline Latzer, Dominic Schmid, Uwe Warnken, Afshin Saffari, Andreas Ziegler, Jennifer Kollmer, Markus Möhlenbruch, Christian Ulfert, Christian Herweh, Brigitte Wildemann, Wolfgang Wick, Markus Weiler |
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| 520 | |a The approved antisense oligonucleotide nusinersen demonstrated convincing clinical results in patients with 5q-linked spinal muscular atrophy (SMA) but predictive biomarkers are lacking to date. | ||
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