Results 71 to 80 of about 4,580 (188)
FEBS Letters, Volume 600, Issue 16, Page 2262-2282, August 2026.We identified a systemic, progressive loss of protein S‐glutathionylation—detected by nonreducing western blotting—alongside dysregulation of glutathione‐cycle enzymes in both neuronal and peripheral tissues of Taiwanese SMA mice. These alterations were partially rescued by SMN antisense oligonucleotide therapy, revealing persistent redox imbalance as ...
Sofia Vrettou, Brunhilde Wirth
wiley +1 more source
Nusinersen Treatment and Healthcare Costs in Spinal Muscular Atrophy
, 2020 Thesis (Master's)--University of Washington, 2020Importance: Patients with severe forms of Spinal Muscular Atrophy (SMA) depend on costly supportive care interventions.
Fox, David
core
Inflammatory markers in cerebrospinal fluid of paediatric spinal muscular atrophy patients receiving nusinersen treatment [PDF]
, 2023 Spinal muscular atrophy (SMA) is a progressive motor neuron disease with onset during infancy or early childhood. Recent therapeutic advances targeting the genetic defect that underlies SMA improved survival in patients with infantile onset SMA (type 1 ...
van der Pol, W. L. +9 more
core
Chemical Biology &Drug Design, Volume 108, Issue 2, August 2026.Multi‐omics (proteomics, metabolomics, and single‐cell/spatial transcriptomics) provides a systems framework to distinguish on‐target efficacy from off‐target and toxicity signatures of chemically modified nucleic acid therapeutics, enabling omics‐guided structure–activity and safety optimisation from discovery to clinical translation. ABSTRACT Nucleic
Gurjit Kaur Bhatti +6 more
wiley +1 more source
Immunization status of patients with spinal muscular atrophy receiving nusinersen therapy
, 2023 Background: Children with chronic neurological diseases, including spinal muscular atrophy (SMA), are particularly susceptible to vaccine-preventable infections.
YİŞ, ULUÇ +5 more
core +1 more source
, 2021 Background!#!Nusinersen is an intrathecally administered antisense oligonucleotide (ASO) that improves motor function in patients with spinal muscular atrophy (SMA).
Hagenacker, Tim +9 more
core +1 more source
Nusinersen in type 0 spinal muscular atrophy: should we treat?
, 2020 A male infant affected by type 0 SMA with one copy of SMN2 received early treatment with Nusinersen at the age of 13 days. He showed mild motor improvement 2 months after treatment started but despite also showing some minimal respiratory ...
de Sanctis R. +9 more
core +1 more source
Orphanet Journal of Rare DiseasesBackground Spinal muscular atrophy (SMA) is a rare neurodegenerative disease that significantly affects multiple systems in children. Nusinersen, the first approved treatment for SMA, enhances SMN protein production by targeting the RNA splicing site of ...
Wenjing Li +3 more
doaj +1 more source
The impact of novel nusinersen treatment on hip stability in spinal muscular atrophy patients
, 2021 Introduction: Hip instability is common in spinal muscular atrophy (SMA) patients due to muscle imbalance. Nusinersen, a novel treatment, has documented effects on improving strength and functionality.
Wong, SHJ, Ip, HHN, Chan, HSS, To, MKT
core
Brain and BehaviorObjective This study was based on a retrospective clinical observational cohort study of a two‐center application of nusinersen in China to evaluate the clinical efficacy and adverse effects of nusinersen in the treatment of SMA (spinal muscular atrophy)
Dan Li +5 more
doaj +1 more source