Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy.

ABSTRACT:

Background

Spinal muscular atrophy (SMA) is a motor neuron disease caused by mutations in the SMN1 gene. Nusinersen, an antisense oligonucleotide, has been shown to improve motor function in SMA patients. However, concerns regarding its renal safety remain as previous studies have linked similar treatments to renal toxicity.

Objective

The aim of this study was to evaluate the effects of the nusinersen treatment on platelet counts and renal functions, specifically urine protein excretion, in SMA patients and to estimate safe urinary protein levels before administration of each intrathecal injection.

Methods

This retrospective study examined data from 33 patients with SMA to assess the effects of nusinersen on motor functions and laboratory parameters including platelet

SUBMITTER: Bahadır Senol H 

PROVIDER: S-EPMC11743982 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature