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Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy
Intrathecal delivery of Nusinersen–an antisense oligonucleotide that promotes survival motor neuron (SMN) protein induction–is an approved therapy for spinal muscular atrophy (SMA). Here, we employed nuclear magnetic resonance (NMR) spectroscopy to longitudinally characterize the unknown metabolic e...
Autores principales: | Errico, Francesco, Marino, Carmen, Grimaldi, Manuela, Nuzzo, Tommaso, Bassareo, Valentina, Valsecchi, Valeria, Panicucci, Chiara, Di Schiavi, Elia, Mazza, Tommaso, Bruno, Claudio, D’Amico, Adele, Carta, Manolo, D’Ursi, Anna Maria, Bertini, Enrico, Pellizzoni, Livio, Usiello, Alessandro |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9599672/ https://www.ncbi.nlm.nih.gov/pubmed/36291640 http://dx.doi.org/10.3390/biom12101431 |
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