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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Dr. Gary Feldman, Principal Investigator for the MRM-3379 Phase 2 clinical trial at the Irvine, California site, standing with an individual with Fragile X syndrome.

Recruiting: Mirum Launches Clinical Trial of MRM-3379 for Fragile X Syndrome

December 1, 2025

Mirum’s MRM-3379 Phase 2 trial builds on FRAXA-funded PDE4D research and is now enrolling males ages 13–45 with Fragile X syndrome. View eligibility and study sites.

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RECONNECT Trial Results: ZYN002 Does Not Meet Primary Endpoint in Fragile X

September 24, 2025

Harmony Biosciences announced that the Phase 3 RECONNECT trial of ZYN002 in Fragile X syndrome failed to meet its primary endpoint.

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Fragile X Research Update: A Turning Point for Treatments and Curative Approaches

September 17, 2025

Fragile X research is at a turning point. FRAXA is funding ASO therapy and CRISPR-based gene reactivation to target the root cause of Fragile X.

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#teamwally shares a joyful outdoor moment supporting Fragile X awareness as the kids explore a steering wheel together

Cognitive Rigidity: a Hallmark of Fragile X and Autism

September 8, 2025

Cognitive rigidity shapes daily life in Fragile X, driving anxiety and repetitive behaviors. Learn its impact on treatment and independence.

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Evaluating Novel Drug Candidates for Fragile X Using the Live Mouse Tracker

September 4, 2025

This grant is funding AI-driven drug discovery, advanced mouse behavior tracking, and gene expression analysis to uncover new treatments for Fragile X syndrome.

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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.

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Shionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome

August 5, 2025

Learn more about Shionogi’s EXPERIENCE clinical trials for adults and adolescents with Fragile X syndrome, FRAXA’s role, and the open-label extension of these trials.

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Sex Differences and the Role of Estrogen Receptors in Fragile X

July 29, 2025

Fragile X syndrome researchers are studying how estrogen receptors shape brain activity and may explain why males and females experience symptoms differently.

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Marine Anais Krzisch, PhD

Role of Microglia in Fragile X Syndrome

June 26, 2025

Learn how Dr. Marine Anais Krzisch’s $35K FRAXA and ASF-funded project uses human iPSC microglia models to uncover pathways for Fragile X syndrome treatment.

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Researcher analyzing gait patterns on a treadmill for Fragile X syndrome motor function study.

Fragile X and Fatigue: Study Reveals How Muscle Function is Altered in Children

April 16, 2025

FRAXA-funded research shows how Fragile X syndrome muscle function affects walking and fatigue, offering insights for future treatments.

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Dr. Thomas Maurin and Dr. Barbara Bardoni, Fragile X researchers and co-authors of a 2025 review on PDE inhibitors published in Cell Reports Medicine.

Fragile X and PDE Inhibitors: A Promising Path Forward for Brain Disorders

March 26, 2025

Fragile X syndrome research shows PDE inhibitors may improve brain function and behavior, with promise for related neurodevelopmental disorders.

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FRAXA-Funded Research Explores ISRIB as a Potential Treatment for Fragile X

February 26, 2025

ISRIB for Fragile X syndrome is being studied as a potential treatment to restore brain function and social behavior. Researchers investigate its effects.

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Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier

August 21, 2024

A $100,000 FRAXA grant to Cincinnati Children’s Hospital is simplifying and testing EEG technology for home use, improving clinical trial accessibility and efficiency.

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Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option

May 20, 2024

Harmony Biosciences’ RECONNECT Phase 3 trial of ZYN002 offered an at-home participation option. Unfortunately the trial reported negative results.

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Inside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments

March 13, 2024

FRAXA-DVI is revolutionizing Fragile X syndrome research, providing efficient, comprehensive and objective preclinical testing of potential treatments.

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Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice

March 6, 2024

Treating Fragile X might require a combination of drugs. FRAXA-DVI tested ibudilast and gaboxadol in Fragile X mice. Together they rescued a wide array of symptoms.

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Unveiling Probiotic Potential in Fragile X Syndrome Clinical Trial

December 20, 2023

First of its kind in Serbia, this clinical trial explores probiotic intervention as a potential treatment avenue for Fragile X syndrome.

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Pharmacological Modulation of Nicotinic Signaling

December 6, 2023

This study tests whether blocking certain nicotine-sensitive receptors in the brain during adolescence can improve attention and cognition in Fragile X.

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Astrocyte Contribution to Sensory Hypersensitivity in Fragile X Syndrome

December 6, 2023

This team studied how faulty calcium signaling in astrocytes contributes to sensory hypersensitivity in Fragile X, aiming to find new astrocyte-targeted treatments.

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SRC Family Kinase Inhibitor as a Potential Treatment for Fragile X Syndrome

May 7, 2023

This $100,000 FRAXA grant will fuel the Smith lab’s new approach to treating Fragile X syndrome using Saracatinib, originally a cancer drug.

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Coming Together for Rare Disease Day 2023

February 28, 2023

Today we mark Rare Disease Day. FRAXA is committed to advancing research on Fragile X, one of the most common rare diseases worldwide.

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Human FMR1 Isoform-Specific Regulation of Translation and Behavior

October 19, 2022

FMRP has multiple forms, and this team will study which isoforms are most important for brain development. This is key for future FMRP replacement therapies.

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Recruiting: Clinical Study of Non-Invasive EEG for Children Ages 2-7

March 5, 2022

Dr. Carol Wilkinson, MD PhD at Boston Children’s Hospital is recruiting children ages 2-7 years with Fragile X syndrome to participate in a study of EEG.

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FRAXA Funded Research

Current Research Grants (44)