FRAXA Q&A Webinar with Dr. Elizabeth Berry-Kravis on the Harmony RECONNECT Trial

Fragile X Research Q&A: Dr. Berry-Kravis on the RECONNECT Trial

Fragile X Research Q&A with Dr. Berry-Kravis on RECONNECT results, placebo effects, outcome measures, methylation, and research directions for families.

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

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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James Fink, Noelle Germain, and Graham Dempsey of Quiver Biosciences

Validate a Novel Antisense Oligonucleotide Therapeutic for Fragile X Syndrome

FRAXA funds Quiver Biosciences to validate a novel antisense oligonucleotide (ASO) therapy for Fragile X syndrome, targeting the root cause of the disorder.

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

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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Dr. Joel Richter and Dr. Sneha Shah in their UMass Chan Medical School lab researching ASO therapy for Fragile X syndrome.

ASO Rescue of FMR1 Mis-Splicing in Neurons and Mitigation of Fragile X Deficits

A new FRAXA grant funds UMass Chan researchers using ASOs in neurons and organoids to correct FMR1 mis-splicing and restore critical FMRP protein.

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Neuronal synapse illustration showing neurotransmitter release, symbolizing how BK channel openers restore balance in Fragile X syndrome.

BK Channel Openers: From FRAXA Seed Funding to Big Pharma Investment and Trials

See how FRAXA’s early grants propelled Fragile X research from lab discoveries to industry momentum, with partners advancing therapies into trials.

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A Tat-Conjugate Approach to Treat Fragile X Syndrome

Explore the Turner Lab’s innovative approach to treating Fragile X using a Tat-linked, truncated FMRP protein designed to restore brain function by replacing the missing protein.

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

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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Metabolic and Mitochondrial Crosstalk in Insulin and cAMP Pathways in Fragile X Syndrome

Fragile X syndrome research explores insulin and cAMP pathway crosstalk, mapping brain metabolism to guide treatment strategies.

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Lida Zoupi and Laura Oliviera with myelin

Oligodendrocytes: a Potential Route to Treat Fragile X Syndrome

This project explores the role of oligodendrocytes in Fragile X. The team will test if improving these cells’ function can restore normal brain activity to treat Fragile X.

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FRAXA Drug Validation Initiative (FRAXA-DVI)

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

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

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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2025 FRAXA Research Grants Webinar with Dr. Michael Tranfaglia and Katie Clapp

Fragile X Syndrome Research in 2025: FRAXA Announces New Grants & Promising Leads

Fragile X syndrome research is advancing in 2025 with new FRAXA-funded grants supporting breakthrough science, therapy platforms, and clinical tools.

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Marine Anais Krzisch, PhD

Role of Microglia in Fragile X Syndrome

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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Jeannie Lee, MD, PhD and Chloe Chen, PhD

Modeling R-Loop Therapy for Fragile X Syndrome in Patient-Derived Brain Organoids

Fragile X syndrome researchers model R-loop therapy in patient-derived brain organoids to restore FMR1, accelerating a curative approach supported by FRAXA.

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UMass Chan Medical School Licenses RNA-Based Therapy to QurAlis, Advancing a New Era in Fragile X Treatment

UMass Chan Medical School licenses RNA-based Fragile X treatment approach using ASOs to QurAlis, moving gene-targeted therapy closer to clinical trials.

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Dr. Jeannie Lee standing in front of a staircase at Harvard Medical School, recipient of the Blavatnik $1M award for Fragile X gene reactivation research

Harvard’s Dr. Jeannie Lee Wins $1M Award to Develop Gene Reactivation Therapy for Fragile X

Dr. Jeannie Lee wins $1M Blavatnik Award to advance FMR1 gene reactivation therapy for Fragile X, building on years of FRAXA-funded research.

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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

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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High-resolution image of dendritic spines—tiny structures on neurons that are altered in Fragile X syndrome.

New Research Targets NMDA Receptor – A Key Player in Brain Communication and Fragile X Syndrome

Fragile X syndrome research identifies NMDA receptor as a promising treatment target, with drugs already in trials offering faster paths to human studies.

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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

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

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

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Urgent Action Needed: Help Secure NIH Funding for Fragile X Research

NIH funding delays threaten Fragile X research, putting critical studies and future treatments at risk. Help us urge Congress and the NIH to act now.

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

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

Current Research Grants (46)