Skip to content

  • Current Fragile X Research Grants
  • Personal Campaigns
  • Request For Applications – Research Grants
FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome

FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome

Accelerating Fragile X Research for Effective Treatments

Donate
  • What is Fragile X?
    • About Fragile X Syndrome
      • What Is Fragile X Syndrome?
      • Symptoms
      • Physical Signs
      • Prevalence and Inheritance
      • Fragile X and Autism
      • Treatment
      • Additional Resources
    • Understanding Fragile X Syndrome
      • What Causes Fragile X Syndrome?
      • The Fragile X Gene
      • Testing/Diagnosis
        • Prenatal Testing
      • Fragile X-Associated Disorders – FXTAS and FXPOI
    • Inspiration
      • #FriendofFRAXA
        • #FriendofFRAXA Submission
      • FRAXA in the News
  • News & Updates
  • Research
    • How Close Are We to a Cure?
    • Curative Therapies Fund
    • Funded Research Grants
    • Fragile X Clinical Trials
    • How to Apply for Research Grants
    • Resources for Researchers
  • Get Involved
    • Stay Connected
      • Attend a Fragile X Event
      • Subscribe to Newsletter
      • Share Your Pictures or Videos With Us
    • Take Action
      • World Fragile X Day
      • Personal Fundraising Campaigns
      • Run Your Own Event
      • Volunteer for FRAXA
        • Advocate for Fragile X
      • FRAXA Shop
    • Participate in Fragile X Research
      • Fragile X Clinical Trials
      • Fragile X Biomarker Research – COMBINEDBrain Consortium
      • Fragile X Clinics
  • About FRAXA
    • Mission Statement
    • Give with Confidence
    • Financial Reports
    • FRAXA in the News
    • Board & Leadership
    • Scientific Advisors
    • FRAXA’s Fragile X Timeline
  • Donate
    • Donate Online
    • Monthly Giving Program
    • Give to a Personal Campaign
    • Other Ways to Give

autism

Role of Microglia in Fragile X Syndrome

FRAXA Research Foundation June 26, 2025June 26, 2025
Marine Anais Krzisch, PhD

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.

Read more

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

FRAXA Research Foundation June 18, 2025
Jeannie Lee, MD, PhD and Chloe Chen, PhD

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

Read more

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

FRAXA Research Foundation March 26, 2025
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 syndrome research shows PDE inhibitors may improve brain function and behavior, with promise for related neurodevelopmental disorders.

Read more

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

FRAXA Research Foundation March 21, 2025May 19, 2025

Shionogi’s EXPERIENCE clinical trials for Fragile X syndrome are nearing completion. Enrollment for the adult trial (EXPERIENCE-301) is now closed, while the adolescent trial (EXPERIENCE-204) is in its final phase. Learn more about the study, FRAXA’s role, and the open-label extension.

Read more

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

FRAXA Research Foundation March 13, 2025March 13, 2025

NIH funding delays are threatening Fragile X research, putting critical studies and future treatments at risk. FRAXA Research Foundation and NFXF are urging Congress and the NIH to act now. Learn how you can help secure funding for Fragile X research.

Read more

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

Anna Devine February 26, 2025March 6, 2025

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

Read more

Callum Cup VIII Scores $19,400 for Fragile X Research – A Milestone Event

Andrew Murphy January 10, 2025January 10, 2025

Callum Cup VIII, Millburn FC’s annual charity match, scored big with $19,400 raised for Fragile X research, bringing total funds to $148,000. Discover how soccer unites for a cause.

Read more

Help Rowan Thrive: Support Fragile X Research

FRAXA Research Foundation December 12, 2024December 15, 2024
Rowan, a young girl with Fragile X syndrome, standing with her family at a scenic outdoor location, symbolizing love and support.

Join the Gale family in supporting Fragile X research through FRAXA. Help Rowan and others thrive by funding life-changing treatments and advancing curative therapies.

Read more

Support the Stevenson Family Campaign

FRAXA Research Foundation November 4, 2024November 6, 2024

The march of time renews the commitment we made to a special needs community 25 years ago. We vowed to dream big and never give up until there were effective treatments available and eventually a cure for Fragile X syndrome, the most commonly inherited cause of intellectual disabilities and autism.

Read more

NPR Spotlights Zatolmilast: A Potential Breakthrough for Fragile X Syndrome

FRAXA Research Foundation October 1, 2024April 21, 2025

NPR spotlights zatolmilast, a promising drug offering new hope for individuals with Fragile X syndrome. Families report life-changing improvements in anxiety, communication, and independence.

Read more

Marvel Biosciences Partners with FRAXA to Test MB204 for Fragile X Syndrome

FRAXA Research Foundation August 20, 2024August 30, 2024

Discover how Marvel Biosciences and FRAXA Research Foundation are collaborating to test MB204, a promising new treatment for Fragile X syndrome, building on groundbreaking adenosine receptor research.

Read more

Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration

Michael Tranfaglia, MD June 24, 2024June 25, 2024

A new treatment target for Fragile X syndrome has emerged from multiple research labs and a pharmaceutical startup. Neurolixis, a FRAXA pharma partner, has announced a new Fragile X development program.

Read more

BK Channel Openers: A New Drug for Fragile X Is Ready for Clinical Trials

Michael Tranfaglia, MD May 31, 2024May 31, 2024

Discover the promising new BK channel opener, SPG601, now entering clinical trials for Fragile X syndrome. Learn about its potential to restore synaptic function and address core symptoms.

Read more

Renewed Hope: Navigating Towards a Cure for Fragile X Syndrome

Theodore Coutilish April 16, 2024April 25, 2024

Discover how Dr. Peter Todd’s latest Fragile X Syndrome research offers hope for advanced treatments and a possible cure, marking a new era in FXS therapy.

Read more

Pharmacologically Activating mGluR7 as a Novel Therapy for Fragile X Syndrome

FRAXA Research Foundation March 7, 2024March 3, 2025

Join Dr. Tsai and Dr. Kumar on a journey into novel treatment avenues for Fragile X syndrome. Learn how activating mGluR7 could be a game-changer, opening up uncharted therapeutic territory.

Read more

Callum Cup’s Big Win: Millburn Charity Soccer Raises $26K for Fragile X Research

Andrew Murphy December 22, 2023December 22, 2023
Callum Cup 2023 Group

Discover the inspiring story of the Callum Cup in Millburn, NJ – a charity soccer match that raised over $26,000 for Fragile X research. Join us in celebrating community spirit and a commitment to a vital cause.

Read more

$100,000 Matching Challenge From The Robert & Ardis James Foundation

FRAXA Research Foundation December 8, 2023December 8, 2023

We are thrilled to announce FRAXA Research Foundation’s most significant and unique matching challenge of the year, thanks to the Robert & Ardis James Foundation. This challenge will help us bring top new talent to Fragile X research.

Read more

Somatosensory Processing as a Therapeutic Target for Fragile X Syndrome

FRAXA Research Foundation September 7, 2023November 19, 2024

Awarded a FRAXA Research grant, Dr. Andrew Stanfield, Dr. Leena E. Williams, and Dr. Damien Wright are set to explore somatosensory processing (sense of touch) in Fragile X syndrome at the University of Edinburgh. Their aim? A noninvasive touch test that could set the stage for future clinical trials in FXS.

Read more

Antisense Oligonucleotides (ASOs) to restore FMRP in Human Fragile X Cerebral Organoids

FRAXA Research Foundation August 24, 2023November 19, 2024

Explore Dr. Richter’s encouraging results with ASOs for Fragile X syndrome. A $100,000 grant now fuels pivotal studies needed to advance toward ASO therapy.

Read more

FRAXA Research Foundation Partners with Autism BrainNet

FRAXA Research Foundation August 17, 2023August 30, 2023

Discover how FRAXA Research Foundation’s collaboration with Autism BrainNet accelerates Fragile X syndrome research by collecting vital postmortem brain tissue. Dive into the significance of brain studies for deeper insights and potential therapeutic interventions.

Read more

ASOs and Fragile X: Addressing the Most Asked Questions

FRAXA Research Foundation August 16, 2023June 25, 2025

Explore the potential of ASOs in treating Fragile X syndrome & FXTAS. Dive into a comprehensive Q&A addressing key questions and breakthrough findings.

Read more

In Memory of Garret

FRAXA Research Foundation August 11, 2023August 14, 2023

Honor Garret Peacock Volker’s extraordinary life by supporting FRAXA’s mission to find a cure for Fragile X syndrome. Your donation can make a difference in the lives of those affected.

Read more

Pioneering Community-Based Drug Development for Fragile X Syndrome

Michael Tranfaglia, MD July 25, 2023July 25, 2023

Discover how FRAXA leverages Community-Based Drug Development to create impactful therapies for Fragile X syndrome. Join us as we reshape the future of rare disease treatment.

Read more

Allos Pharma Advances Phase 3 Clinical Trial Design for Potential Fragile X Syndrome Treatment, Arbaclofen

FRAXA Research Foundation May 24, 2023May 24, 2023

Discover Allos Pharma’s advancements in a pivotal Phase 3 trial for Fragile X syndrome treatment, Arbaclofen. Learn how their FDA-informed trial design might finally bring hope to the Fragile X community.

Read more

Breakthrough Discoveries in Fragile X Research: Insights from Special Banbury Meeting on Curative Therapies

Michael Tranfaglia, MD May 18, 2023May 18, 2023
2023 Fragile X Research Banbury Meeting (1)

Explore the latest breakthroughs in Fragile X research unveiled at the recent Banbury Meeting. Discover novel strategies, from gene therapy to protein replacement, that bring hope for curative therapies.

Read more
  • ← Previous

FRAXA Research Foundation

  • 10 Prince Place, Suite 203
    Newburyport, MA 01950-2657
  • info@fraxa.org
  • (978) 462-1866
Donate Online
GuideStar_Exchange-Platinum_Participant
GreatNonprofits 2024 top rated award - FRAXA Research Foundation
charity_navigator-4_star_charity

Join our Newsletter Mailing List

Name*
This field is for validation purposes and should be left unchanged.

Copyright © 2025 FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome
FRAXA Research Foundation is a registered 501(c)3 organization.
Tax ID: 04-3222167

Change Privacy Settings

  • Privacy Policy
  • Cookie Policy
  • About Fragile X
  • Ways to Give