Clinical Research Directory

Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA

Sponsor: Istituto Giannina Gaslini

Summary

Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder caused by mutations in the SMN1 gene, leading to motor neuron degeneration and progressive muscle atrophy. Despite recent advances in therapeutic options, the clinical management of SMA remains challenging due to the heterogeneity of disease progression and variability in treatment response. This study aims to identify and validate novel biomarkers to improve the diagnosis, prognosis, and therapeutic monitoring of patients with SMA through a multi-omics and translational research approach.

Official title: Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA - Approcci Avanzati Multi-omici e Sistemici Per Identificare Nuovi Biomarcatori Per la SMA

Key Details

Conditions

Locations (3)