Longitudinal Transcriptomic Analysis Reveals Systemic Effects of Risdiplam in Adults with Spinal Muscular Atrophy

Background: Spinal Muscular Atrophy (SMA) is a neurodegenerative disease caused by reduced survival motor neuron (SMN) protein levels due to <i>SMN1</i> gene mutations. The natural history of SMA has dramatically changed since innovative therapies were approved; among them, Risdiplam (an oral molecu...

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Bibliografische Detailangaben

Hauptverfasser:	Maria Liguori, Arianna Consiglio, Eustachio D’Errico, Ylenia Antonacci, Martina Coffa, Alessandro Introna, Isabella Laura Simone
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	MDPI AG 2026-06-01
Schriftenreihe:	Brain Sciences
Schlagworte:	spinal muscular atrophy Risdiplam Transcriptomics <i>SMN2</i> gene RNA sequencing
Online-Zugang:	https://www.mdpi.com/2076-3425/16/6/643
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