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Circulating transcriptional biomarkers serve as surrogates of disease progression in Charcot-Marie-Tooth 1A disease

2025-09-02

Abstract excerpt

<h4>Objective</h4> Charcot-Marie-Tooth disease type 1A (CMT1A), the most common inherited peripheral neuropathy, results from PMP22 gene duplication and presents with progressive motor and sensory deficits. Despite well-defined genetics, therapeutic development is hindered by the lack of sensitive clinical outcome assessments (CAO) given slow disease progression and variable severity. This study aimed to identify...

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Literature Corpus work
f8ac6ef1-4149-5f01-9a2c-371e8eda5eb4
DOI
10.1101/2025.08.28.25334434
Open publication

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Circulating transcriptional biomarkers serve as surrogates of disease progression in Charcot-Marie-Tooth 1A diseaseDOI 10.1101/2025.08.28.25334434
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