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