Cerebrospinal fluid characteristics of patients treated with intrathecal nusinersen for spinal muscular atrophy.

ABSTRACT:

Introduction/aims

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.

Methods

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).

Results

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

SUBMITTER: Orbach R 

PROVIDER: S-EPMC9828332 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature