Blood-Based Biomarkers Predictive of Metformin Target Engagement in Fragile X Syndrome.

ABSTRACT: Recent advances in neurobiology have provided several molecular entrees for targeted treatments for Fragile X syndrome (FXS). However, the efficacy of these treatments has been demonstrated mainly in animal models and has not been consistently predictive of targeted drugs' response in the preponderance of human clinical trials. Because of the heterogeneity of FXS at various levels, including the molecular level, phenotypic manifestation, and drug response, it is critically important to identify biomarkers that can help in patient stratification and prediction of therapeutic efficacy. The primary objective of this study was to assess the ability of molecular biomarkers to predict phenotypic subgroups, symptom severity, and treatment response to metformin in clinically treated patients with

SUBMITTER: Jasoliya M 

PROVIDER: S-EPMC7349631 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature