Article

ROC Analysis of Biomarker Combinations in Fragile X Syndrome-Specific Clinical Trials: Evaluating Treatment Efficacy via Exploratory Biomarkers

2025-05-29

Abstract excerpt

Fragile X Syndrome (FXS) is a rare neurodevelopmental disorder caused by a trinucleotide repeat expansion on the 5’ untranslated region of the FMR1 gene. FXS is characterized by intellectual disability, anxiety, sensory hypersensitivity, and difficulties with executive function. A recent phase 2 placebo-controlled clinical trial assessing BPN14770, a first-in-class phosphodiesterase 4D allosteric inhibitor, in 30...

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Literature Corpus work: 9135d01c-853b-5523-ba99-80ea11718e29
DOI: 10.1101/2025.05.29.25328581

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