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...

وصف كامل

التفاصيل البيبلوغرافية
الحاوية / القاعدة:Brain Sciences
المؤلفون الرئيسيون: Maria Liguori, Arianna Consiglio, Eustachio D’Errico, Ylenia Antonacci, Martina Coffa, Alessandro Introna, Isabella Laura Simone
التنسيق: مقال
اللغة:الإنجليزية
منشور في: MDPI AG 2026-06-01
الموضوعات:
spinal muscular atrophy
Risdiplam
Transcriptomics
<i>SMN2</i> gene
RNA sequencing
الوصول للمادة أونلاين:https://www.mdpi.com/2076-3425/16/6/643

الانترنت

https://www.mdpi.com/2076-3425/16/6/643

مواد مشابهة