{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["66(6)"],"submitter":["Orbach R"],"pubmed_abstract":["<h4>Introduction/aims</h4>There is limited information on the potential effects of repeated intrathecal antisense oligonucleotide drug delivery on cerebrospinal fluid (CSF) biochemical and blood cell profiles. This study aimed to examine longitudinal changes in the biochemical components (glucose, protein) and blood cell counts in the CSF of spinal muscular atrophy (SMA) patients treated with intrathecal nusinersen.<h4>Methods</h4>We collected and analyzed clinical and CSF parameters (cell count, protein, glucose, culture) of 50 individuals with SMA during nusinersen treatment (22 type 1, 17 type 2, and 11 type 3).<h4>Results</h4>The median protein concentration at baseline and during treatment was within the normal range but rose during treatment and was significantly above baseline at th"],"journal":["Muscle & nerve"],"pagination":["762-766"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC9828332"],"repository":["biostudies-literature"],"pubmed_title":["Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy."],"pmcid":["PMC9828332"],"pubmed_authors":["Sadot E","Sagi L","Zisberg T","Tokatly Latzer I","Fattal-Valevski A","Orbach R","Shtamler A"],"additional_accession":[]},"is_claimable":false,"name":"Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy.","description":"<h4>Introduction/aims</h4>There is limited information on the potential effects of repeated intrathecal antisense oligonucleotide drug delivery on cerebrospinal fluid (CSF) biochemical and blood cell profiles. This study aimed to examine longitudinal changes in the biochemical components (glucose, protein) and blood cell counts in the CSF of spinal muscular atrophy (SMA) patients treated with intrathecal nusinersen.<h4>Methods</h4>We collected and analyzed clinical and CSF parameters (cell count, protein, glucose, culture) of 50 individuals with SMA during nusinersen treatment (22 type 1, 17 type 2, and 11 type 3).<h4>Results</h4>The median protein concentration at baseline and during treatment was within the normal range but rose during treatment and was significantly above baseline at th","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Dec","modification":"2026-05-29T21:09:44.25Z","creation":"2025-04-05T10:26:43.917Z"},"accession":"S-EPMC9828332","cross_references":{"pubmed":["36214191"],"doi":["10.1002/mus.27731"]}}