Preliminary insights into RNA in CSF of pediatric SMA patients after 6 months of nusinersen

Abstract Background Spinal muscular atrophy (SMA) is a rare autosomal-recessive neurodegenerative disorder caused by mutations in survival motor neuron 1 (SMN1) gene, and consequent loss of function of SMN protein, which results in progressive loss of lower motor neurons, and muscular wasting. Antis...

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Bibliographic Details

Published in:	Biology Direct
Main Authors:	M. Garofalo, S. Bonanno, S. Marcuzzo, C. Pandini, E. Scarian, F. Dragoni, R. Di Gerlando, M. Bordoni, S. Parravicini, C. Gellera, R. Masson, C. Dosi, R. Zanin, O. Pansarasa, C. Cereda, A. Berardinelli, S. Gagliardi
Format:	Article
Language:	English
Published:	BMC 2023-09-01
Subjects:	Spinal muscular atrophy Nusinersen Cell-free RNA Transcriptomics
Online Access:	https://doi.org/10.1186/s13062-023-00413-6