Baclofen-associated neurophysiologic target engagement across species in fragile X syndrome.

ABSTRACT:

Background

Fragile X syndrome (FXS) is the most common inherited form of neurodevelopmental disability. It is often characterized, especially in males, by intellectual disability, anxiety, repetitive behavior, social communication deficits, delayed language development, and abnormal sensory processing. Recently, we identified electroencephalographic (EEG) biomarkers that are conserved between the mouse model of FXS (Fmr1 KO mice) and humans with FXS.

Methods

In this report, we evaluate small molecule target engagement utilizing multielectrode array electrophysiology in the Fmr1 KO mouse and in humans with FXS. Neurophysiologic target engagement was evaluated using single doses of the GABA_B selective agonist racemic baclofen (RBAC).

Results

In Fmr1 KO mice a

SUBMITTER: Jonak CR 

PROVIDER: S-EPMC9513876 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature