Article

Large-scale Proteomics Profiling of Peripheral Blood of DM1 patients identifies biomarkers for disease severity and functional capacity

2025-09-08

Abstract excerpt

<h4>Background: </h4> Myotonic Dystrophy Type 1 (DM1), the most common genetic neuromuscular disorder in adults, poses significant challenges for drug development due to its multisystem nature and high clinical variability in symptoms and disease progression. With a growing number of therapies entering clinical trials, this study addresses the urgent need for biomarkers that can serve as surrogate endpoints. <h4>M...

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Literature Corpus work: ad442a6a-c8a6-5005-a4ea-05ad4c603453
DOI: 10.1101/2025.09.05.25335077

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