ERK Archives

Enhancement of NMDA Receptor Signaling for the Treatment of Fragile X Syndrome

June 15, 2025

Drs. Emily Osterweil and Stephanie Barnes investigated NMDA receptor signaling and how rebalancing protein synthesis could correct Fragile X brain abnormalities.

Validating Novel Inhibitors of ERK Signalling to Treat Fragile X Syndrome

February 13, 2023

This team is testing ERK inhibitors — drugs that may calm overactive brain signaling in Fragile X. Early results in mice show strong benefits with minimal side effects.

Clinical Trial of Metformin for Fragile X Syndrome

October 24, 2021

FRAXA-funded open-label trial found that metformin led to increased GABA-mediated cortical inhibition, suggesting metformin modulates core Fragile X pathways.

Brain Organoids and Therapeutic Development for Fragile X and Other Rare Diseases

July 15, 2020

In this webinar we hear from Alysson R. Muotri, PhD, Professor at University of California San Diego Stem Cell Program and Fabio C. Tucci, PhD, CEO and co-founder at Epigen Biosciences.

Effects of Metformin in Fmr1 Knockout Mouse Model of Fragile X Syndrome

November 8, 2017

Metformin, a safe diabetes drug, activates AMPK to rebalance protein synthesis. FRAXA-funded work investigated its potential to treat Fragile X.

18th International Fragile X and Related Neurodevelopmental Disorders Workshop, Quebec, Canada

In Their Own Words: Reports From the International Fragile X Workshop

October 29, 2017

The 18th International Fragile X Workshop in Quebec was a great success, featuring more Fragile X research than ever before!

Dr. Tom Jongens and Dr. Sean McBride study Fragile X Fruit Flies

Fruit Flies to Model and Test Fragile X Treatments

September 13, 2015

Boosting cAMP signaling restores memory and fixes brain-signaling defects in Fragile X models, suggesting diabetes drugs like metformin may help.

Social Behavior as an Outcome Measure for Fragile X Clinical Trials

September 13, 2013

FRAXA funding helped identify reliable social behavior tests in Fragile X mice and showed an mGluR5 treatment could improve sociability, guiding future trials.

Lovastatin Discovery in Fragile X Mice Leads FRAXA to Fund Clinical Trials

June 11, 2013

FRAXA honored Dr. Emily Osterweil for discovering that lovastatin can correct key Fragile X abnormalities. Her findings were published in Neuron.

What Works, and What Doesn’t

September 7, 2012

Early on, no one knew which path would work. Now the results are clear, and they’re directing FRAXA toward the next major Fragile X treatment breakthrough.