Drs. Oswald Steward and Patricia Pirbhoy in their laboratory, studying RNA localization and gene activity in Fragile X syndrome using spatial transcriptomics.

Uncovering the Dynamics of FMR1 Using Spatial Transcriptomics for Fragile X

December 12, 2025

Scientists investigate how changes in RNA localization affect cognition and behavior in Fragile X, building knowledge toward more precise therapies.

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The Endocannabinoid System and Fragile X Syndrome

July 6, 2024

This project will examine how CBD and other drugs targeting the endocannabinoid system affect hyperexcitable Fragile X neurons to identify new treatment strategies.

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Targeting Cognitive Function in Fragile X Syndrome

February 14, 2023

Why do males and females experience Fragile X differently? This team is studying brain signaling pathways to uncover sex-based differences and guide treatments.

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Transcriptional Signatures Sensitive to Cognition-Improving Pharmacological Treatments in Fragile X Syndrome

June 27, 2022

This team is defining Fragile X “molecular signatures” to use as biomarkers. They’ll test CBD and other drugs in mice and compare findings to human brain data.

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Cannabinoids as a Treatment for Fragile X Syndrome

November 4, 2021

This team uses EEG to study sensory hypersensitivity in Fragile X. By testing drugs in mice, they aim to find treatments that calm brain overactivity.

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2021 Fragile X Research Grants Funded by FRAXA Research Foundation

October 1, 2021

Each year, FRAXA funds a diverse portfolio of research. FRAXA Fellowships and Grants are seed funding for the future, the feedstock for the Fragile X treatment development pathway.

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Link Between Lipid Profile, eCBome System and Gut Microbiome in Fragile X Syndrome

July 15, 2021

Why does obesity challenge so many people with Fragile X? Dr. Caku’s team has found that Fragile X syndrome causes changes in the tiny organisms that live in our gut.

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Pivotal Phase 3 Trial of Zygel in Severe Fragile X Possible This Year

May 17, 2021

Zynerba Pharmaceuticals received advice from the U.S. Food and Drug Administration (FDA) on the design of a Phase 3 clinical trial of Zygel as a cannabidiol treatment for Fragile X syndrome.

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Pharmacotherapeutic Effects of Cannabidiol (CBD) in Fragile X syndrome (FXS) and Autism Spectrum disorder (ASD)

May 6, 2021

This study tested CBD (cannabidiol) treatment in male and female Fragile X mice to learn how and why it works and whether gender affects responses to CDB treatment.

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Cellular-Specific Therapeutic Targeting of Inhibitory Circuits in Fragile X Syndrome

April 21, 2021

The team studied how inhibitory brain circuits malfunction in Fragile X and tested ways to restore balance by targeting mGluR and endocannabinoid signaling.

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Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest

October 22, 2020

Zynerba presented clinical trial results for Zygel at a recent neurology conference. Zygel, an experimental cannabidiol (CBD) gel, may reduce behavioral abnormalities in children with Fragile X syndrome who have more severe disease.

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Devon Binder, PhD; Iryna Ethell, PhD, Patricia Pirbhoy, PhD, at UC Riverside School of Medicine

Understanding and Reversing Hypersensitivity to Sounds in Fragile X Syndrome

June 20, 2018

This FRAXA grant studied why people with Fragile X are overly sensitive to sound and tested drug strategies to calm the brain’s overactive auditory circuits.

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Bradley Alger, PhD

The Endocannabinoid System in a Mouse Model of Fragile X Syndrome

March 4, 2015

Fragile X disrupts endocannabinoid signaling. This study in mice demonstrated that correcting it may calm brain hyperexcitability and improve symptoms.

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Andres Ozaita, PhD

Targeting the Endocannabinoid System in Adult Fragile X Mice

September 15, 2014

CB1 blockade with rimonabant reversed cognitive, sensory, and seizure symptoms in FXS mice, highlighting the endocannabinoid system as a therapeutic target.

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Dysruption of Endocannabinoid Mediated Synaptic Plasticity in the Nac of FMR1 Null Mice

June 10, 2013

This team was able to establish the endocannabinoid system as a therapeutic target for Fragile X syndrome. They also showed that the system can be targeted with pharmacology.

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Endocannabinoid Mediated Synaptic Plasticity in Fragile X Mice

June 2, 2013

FRAXA-funded studies found faulty endocannabinoid signaling in Fragile X brain circuits for reward and emotion, and boosting 2-AG restored normal function.

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FRAXA Funded Research

Current Research Grants (37)