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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...

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Detalles Bibliográficos
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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
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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