Identification of Novel Biomarkers of Spinal Muscular Atrophy and Therapeutic Response by Proteomic and Metabolomic Profiling of Human Biological Fluid Samples

Identification of Novel Biomarkers of Spinal Muscular Atrophy and Therapeutic Response by Proteomic and Metabolomic Profiling of Human Biological Fluid Samples

Spinal muscular atrophy (SMA) is a neuromuscular disease resulting from mutations or deletions in <i>SMN1</i> that lead to progressive death of alpha motor neurons, ultimately leading to severe muscle weakness and atrophy, as well as premature death in the absence of treatment. Recent ap...

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Bibliographic Details
Main Authors: Megi Meneri, Elena Abati, Delia Gagliardi, Irene Faravelli, Valeria Parente, Antonia Ratti, Federico Verde, Nicola Ticozzi, Giacomo P. Comi, Linda Ottoboni, Stefania Corti
Format: Article
Language:English
Published: MDPI AG 2023-04-01
Series:Biomedicines
Subjects:
antisense oligonucleotide
cerebrospinal fluid
proteome
metabolome
nusinersen
spinal muscular atrophy
Online Access:https://www.mdpi.com/2227-9059/11/5/1254

Internet

https://www.mdpi.com/2227-9059/11/5/1254

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