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

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第一作者： Maria,Liguori

第一单位： National Research Council, Department of Biomedicine, Institute of Biomedical Technologies, Bari Unit, 70125 Bari, Italy.

作者： Maria,Liguori ^[1] ; Arianna,Consiglio ^[1] ; Eustachio,D'Errico ^[2] ; Ylenia,Antonacci ^[3] ; Martina,Coffa ^[1] ; Alessandro,Introna ^[2] ; Isabella Laura,Simone ^[4]

作者单位： National Research Council, Department of Biomedicine, Institute of Biomedical Technologies, Bari Unit, 70125 Bari, Italy. ^[1] Neurology Unit, Department of Translational Biomedicine and Neuroscience, University of Bari "Aldo Moro", 70124 Bari, Italy. ^[2] National Research Council, Department of Biomedicine, Institute of Biomedical Technologies, Bari Unit, 70125 Bari, Italy.;National Research Council, Institute of Biomembranes, Bioenergetics and Molecular Biotechnologies, 70125 Bari, Italy. ^[3] School of Medicine, University of Bari "Aldo Moro", 70124 Bari, Italy. ^[4]

关键词 RNA sequencing Risdiplam SMN2 gene Transcriptomics spinal muscular atrophy

DOI 10.3390/brainsci16060643

PMID 42352652

发布时间 2026-06-26

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2026年16卷6期

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

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

Brain sciences

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