2015 Grants Archives

FRAXA Drug Validation Initiative (FRAXA-DVI)

August 13, 2025

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing to validate investigational and repurposed compounds for Fragile X.

Lovamix: Clinical Trial of Combined Treatment of Minocycline and Lovastatin in Fragile X Syndrome

February 3, 2022

FRAXA funded the LovaMiX trial of lovastatin + minocycline for Fragile X. 2022 results show safety and support continued study of this dual-target treatment approach.

Novel Modulators of Potassium Channels to Treat Fragile X

January 2, 2019

FRAXA-funded Yale research showed disrupted Kv3.1 and Slack potassium channels impair neuronal timing in Fragile X. Published findings support Kv3.1 as a treatment target.

Mechanisms of Tolerance to Chronic mGluR5 Inhibition

September 19, 2017

FRAXA supported research showing mGluR5 antagonist tolerance develops quickly in Fragile X models, guiding new strategies to prevent or overcome it.

Frank Kooy, PhD, at University of Antwerp

Clinical Trial of Ganaxolone in Patients with Fragile X Syndrome

September 2, 2017

Dr. Frank Kooy and colleagues conducted a double blind crossover trial of ganaxolone in patients with Fragile X with FRAXA funding. Results of this study were mixed.

Biomarker Discovery and Validation for Fragile X Syndrome

March 24, 2017

This grant supported discovery of protein-based biomarkers for Fragile X to create objective outcome measures that translate from mouse studies to human trials.

Function of FMRP and Test of a Novel Therapeutic Approach in a Fragile X Mouse Model

September 16, 2016

FRAXA-supported work has identified DgkK as a critical enzyme lost in Fragile X. Drugs that raise DgkK levels may correct brain signaling and improve symptoms.

Correcting Defects in Astrocyte Signaling in Fragile X Syndrome

September 14, 2016

Astrocytes, brain cells which support neurons, do not transmit signals. Fragile X treatment strategies have been proposed based on correction of “astrocyte phenotypes”.

Sensory Hypersensibility in Fragile X Syndrome and BK Channel Openers

September 12, 2016

With $366,100 in FRAXA funding, researchers tested BK channel–opening drugs to fix sensory abnormalities in Fragile X mice; early results showed broad behavioral rescue.

Fragile X Mutant Mouse Models

September 10, 2016

With $375,000 in grants from FRAXA, Dr. David Nelson developed an array of advanced mouse models of Fragile X. These models are available at Jackson Labs (JAX).

MicroRNAs as Biomarkers in Fragile X Syndrome

September 10, 2016

The team at Johns Hopkins University studied groups of small RNAs, known as microRNAs, which are greatly decreased in brain tissue of Fragile X mice vs. normal controls.

Repurposing Drugs to Dampen Hyperactive Nonsense-Mediated Decay in Fragile X Syndrome

September 8, 2016

FRAXA-funded research showed nonsense-mediated mRNA decay is overactive in Fragile X, pointing to existing NMD-suppressing drugs like caffeine as potential treatments.

Abnormalities of Synaptic Plasticity in the Fragile X Amygdala

February 22, 2016

With FRAXA funding, Dr. Sumantra Chattarji at NCBS explored how Fragile X alters amygdala function. Results were published.

Peter Vanderklish, PhD, at Scripps Research Institute, FRAXA research grant

Targeting AMP-Activated Protein Kinase Pathway in Fragile X Syndrome

September 13, 2015

With this grant from FRAXA, Dr. Peter Vanderklish explored AMPK activators to treat Fragile X. Both metformin and resveratrol, found in red wine, are AMPK activators.

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.

Margaret King accepted the FRAXA Pioneer Award on behalf of Dr. Richard Jope, at the 2013 FRAXA Investigators Meeting

Analysis of Developmental Brain Dysfunction in Families

September 12, 2015

No strong behavioral similarities were found between parents and children with Fragile X, indicating family history may not guide clinical trial recruitment.

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

April 8, 2015

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

Paul Lombroso, PhD, Yale University, FRAXA Investigator

Inhibitors of STEP as a Novel Treatment of Fragile X Syndrome

February 26, 2015

STEP inhibition reversed behavioral and synaptic Fragile X deficits in mice (Neuropharmacology, 2018), highlighting STEP as a promising treatment target.

Molecular Mechanisms of Cytoskeletal Regulation by FMRP

January 9, 2015

With FRAXA funding, Dr. Jaffrey linked FMR1 loss to abnormal dendritic spines via RhoA signaling, suggesting RhoA-targeted therapies could help treat Fragile X.

FRAXA Drug Validation Initiative (FRAXA-DVI)

Lovamix: Clinical Trial of Combined Treatment of Minocycline and Lovastatin in Fragile X Syndrome

Novel Modulators of Potassium Channels to Treat Fragile X

Mechanisms of Tolerance to Chronic mGluR5 Inhibition

Clinical Trial of Ganaxolone in Patients with Fragile X Syndrome

Biomarker Discovery and Validation for Fragile X Syndrome

Function of FMRP and Test of a Novel Therapeutic Approach in a Fragile X Mouse Model

Correcting Defects in Astrocyte Signaling in Fragile X Syndrome

Sensory Hypersensibility in Fragile X Syndrome and BK Channel Openers

Fragile X Mutant Mouse Models

MicroRNAs as Biomarkers in Fragile X Syndrome

Repurposing Drugs to Dampen Hyperactive Nonsense-Mediated Decay in Fragile X Syndrome

Abnormalities of Synaptic Plasticity in the Fragile X Amygdala

Targeting AMP-Activated Protein Kinase Pathway in Fragile X Syndrome

Fruit Flies to Model and Test Fragile X Treatments

Analysis of Developmental Brain Dysfunction in Families

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

Inhibitors of STEP as a Novel Treatment of Fragile X Syndrome

Molecular Mechanisms of Cytoskeletal Regulation by FMRP

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