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

<b>Background:</b> 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 p...

Descripción completa

Detalles Bibliográficos

Autores principales:	Svenja Neuhoff, Linda-Isabell Schmitt, Kai Christine Liebig, Stefanie Hezel, Nick Isana Tilahun, Christoph Kleinschnitz, Markus Leo, Tim Hagenacker
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	MDPI AG 2025-07-01
Colección:	Biomedicines
Materias:	spinal muscular atrophy biomarker NPTX2 nusinersen
Acceso en línea:	https://www.mdpi.com/2227-9059/13/8/1821
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!