Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients

BACKGROUND AND PURPOSE: The antisense oligonucleotide nusinersen (Spinraza) regulates splicing of the survival motor neuron 2 (SMN2) messenger RNA to increase SMN protein expression. Nusinersen has improved ventilator‐free survival and motor function outcomes in infantile onset forms of spinal muscu...

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Detalles Bibliográficos
Autores principales: Magen, Iddo, Aharoni, Sharon, Yacovzada, Nancy Sarah, Tokatly Latzer, Itay, Alves, Christiano R. R., Sagi, Liora, Fattal‐Valevski, Aviva, Swoboda, Kathryn J., Katz, Jacob, Bruckheimer, Elchanan, Nevo, Yoram, Hornstein, Eran
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Rare Neurological Conditions
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9544362/
https://www.ncbi.nlm.nih.gov/pubmed/35510740
http://dx.doi.org/10.1111/ene.15382

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