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Cerebrospinal Fluid Proteomic Changes after Nusinersen in Patients with Spinal Muscular Atrophy
Disease-modifying treatments have transformed the natural history of spinal muscular atrophy (SMA), but the cellular pathways altered by SMN restoration remain undefined and biomarkers cannot yet precisely predict treatment response. We performed an exploratory cerebrospinal fluid (CSF) proteomic st...
Autores principales: | Beaudin, Marie, Kamali, Tahereh, Tang, Whitney, Hagerman, Katharine A., Dunaway Young, Sally, Ghiglieri, Lisa, Parker, Dana M., Lehallier, Benoit, Tesi-Rocha, Carolina, Sampson, Jacinda B., Duong, Tina, Day, John W. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10607664/ https://www.ncbi.nlm.nih.gov/pubmed/37892834 http://dx.doi.org/10.3390/jcm12206696 |
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