Article

Neuronal Pentraxin 2 as a Potential Biomarker for Nusinersen Therapy Response in Adults with Spinal Muscular Atrophy: A Pilot Study

2025-06-30

Abstract excerpt

<h4>Background: </h4> The treatment landscape for spinal muscular atrophy (SMA) has changed significantly with the approval of gene-based therapies such as nusinersen for adults with SMA (pwSMA). Despite their efficacy, high costs and treatment burden highlight the need for biomarkers to objectify or predict treatment response. This study aimed to identify such biomarkers. <h4>Methods:</h4> A proteomic analysis of...

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Literature Corpus work: 9578a28e-6ea1-523a-8463-b76c9d48f845
DOI: 10.20944/preprints202506.2242.v1

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