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

Developmental Motor Phenotype in Fragile X Syndrome

July 12, 2025

A little known sign of Fragile X is unsteady walking. This team is developing outcome measures of gait for future treatment trials. Results published.

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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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#teamwally gathered in matching Fragile X shirts for a group photo during a family fundraiser

#teamwally: Help Find a Cure for Fragile X Syndrome

May 9, 2025

Join #teamwally in supporting Fragile X research. Read Wally’s inspiring journey and help fund a future of hope and breakthroughs for kids like him.

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Stephanie Barnes, PhD

Enhancing NMDA Receptor Signaling to Treat Fragile X Syndrome

May 7, 2025

FRAXA-backed work revealed NMDA receptors may hold the key to correcting brain signaling in Fragile X, pointing to new treatment strategies.

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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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Combining Gene Reactivation with Controlled Sound Exposure to Treat Fragile X Syndrome

March 11, 2025

The team combined gene reactivation with noninvasive acoustic stimulation in a mouse model as an approach to treating Fragile X syndrome.

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Advancing Curative Therapy for Fragile X Syndrome: Turner Lab Secures $1M Grant

March 10, 2025

Fragile X syndrome research accelerates as the Turner Lab secures $1M to expand FRAXA-funded work on curative therapy targeting the missing FMRP protein.

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

February 5, 2025

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

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GEXVal and FRAXA Collaborate to Advance Fragile X Research with Phase 2a Trial

January 2, 2025

GEXVal and FRAXA collaborate to advance Fragile X research with the Phase 2a trial of GXV-001, supported by AMED’s funding program.

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BK Channel Openers: A New Hope for Fragile X Treatment – Insights from Kaerus Bioscience CEO Robert Ring

October 22, 2024

Kaerus Bioscience’s BK channel openers for Fragile X syndrome are advancing through Phase 1 trials, offering hope for new treatments with FRAXA’s continued support.

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Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

October 9, 2024

This Stanford University team assessed combinatorial drug treatments to correct a broad spectrum of deficits observed in Fragile X syndrome. Results published.

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Investigating the Role of the Insulin Degrading Enzyme (IDE) in Fragile X Syndrome

October 7, 2024

This study explores how disrupted insulin signaling affects metabolism and brain function in Fragile X, revealing new treatment targets for both body and mind.

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Understanding Anxiety in Fragile X: Insights from Dr. Carrie Buchanan’s Webinar

August 1, 2024

Dr. Carrie Buchanan shares strategies to manage anxiety in individuals with Fragile X syndrome, including non-pharmacological treatments and medication options.

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To Interrogate the Developmental Timing for Treating Fragile X Syndrome

July 29, 2024

Are there critical periods in Fragile X syndrome? Will treatment work in adults as well as in children? This team aims to answer these questions.

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Preclinical Testing of High Fat/Low Carb Diets in Fragile X Mice and Cells

July 28, 2024

Dr. Cara Westmark’s team will use mice to determine if palatable Atkins-type diets can improve sleep and boost learning skills for those with Fragile X syndrome.

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Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration

June 24, 2024

FRAXA pharma partner Neurolixis has filed patents for treatment of Fragile X with a highly selective new drug, NLX-101, which targets the serotonin receptor 5-HT1A.

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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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Pharmacologically Activating mGluR7 as a Novel Therapy for Fragile X Syndrome

March 7, 2024

Join Dr. Tsai and Dr. Kumar on a journey into novel treatments for Fragile X syndrome. Activating mGluR7 could be a game-changer, opening up uncharted therapeutic territory.

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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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FRAXA Funded Research

Current Research Grants (38)