Group photo of Callum Cup IX participants gathered at Millburn High School during the annual charity soccer match supporting Fragile X research.

Callum Cup IX Brings Community Together, Raising $20,000 for Fragile X Research

December 17, 2025

Callum Cup IX brought the Millburn FC community together, raising $20,000 for the FRAXA Research Foundation in support of Fragile X research.

Read More »

FRAXA Drug Validation Initiative (FRAXA-DVI)

December 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.

Read More »
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.

Read More »

Shionogi Shares Update on Zatolmilast Fragile X Clinical Trials

October 27, 2025

Shionogi shares an update on its zatolmilast Fragile X clinical trials, outlining progress in U.S. studies and next steps in data analysis and evaluation.

Read More »
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

October 2, 2025

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

Read More »

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.

Read More »

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.

Read More »

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.

Read More »

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

January 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!

Read More »

NPR Spotlights Zatolmilast: A Potential Breakthrough for Fragile X Syndrome

October 1, 2024

NPR spotlights zatolmilast, a promising investigational drug to treat Fragile X syndrome. Families report life-changing improvements in learning and independence.

Read More »

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.

Read More »

Fragile X Clinical Trial of New PDE4D Inhibitor from Tetra

November 20, 2022

A $200K FRAXA grant enabled a successful Phase 2 trial of a PDE4D inhibitor for adult men with Fragile X, showing strong cognitive gains without side effects or tolerance.

Read More »

Positive Results Reported in Phase II Fragile X Clinical Trial of PDE4D Inhibitor Zatolmilast from Tetra Therapeutics

November 2, 2020

Today, Tetra Therapeutics announces the first unequivocally positive phase 2 clinical trial in Fragile X syndrome, press release below. The results do not depend on carving out a subset of patients or post hoc analysis.

Read More »

Companies Move to Advance Potential Cognitive Treatment for Fragile X

March 13, 2020

Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment candidate for disorders marked by cognitive and memory deficits, including Fragile X syndrome and Alzheimer’s disease. The agreement builds on an earlier collaboration between the two companies, and aims to further accelerate BPN14770’s development and potential marketing. It is currently in clinical testing in both Fragile X and Alzheimer’s patients.

Read More »

Tetra Announces $40M to Advance BPN14770 for FXS and Alzheimer’s Disease

December 19, 2018

Tetra Discovery Partners has signed a multi-part deal that could bring it up to $160 million, plus royalties, from Shionogi & Co, Ltd, a Japanese major research-driven pharmaceutical company. Tetra currently is conducting an investigational Phase 2 study of BPN14770 in adults with Fragile X Syndrome, an indication for which BPN14770 has received Orphan Drug Designation from the US Food and Drug Administration. This clinical trial was made possible by early work with the FRAXA-DVI and over $200,000 from FRAXA.

Read More »
Funding opportunities - FRAXA investigators

Combinatorial Drug Treatment in a Model of Fragile X Syndrome using Novel Biomarkers

October 3, 2017

University of California researchers Khaleel Razak, PhD, and Jonathan W. Lovelace, PhD, explored drug combinations to limit hypersensitivity to sounds in Fragile X mice.  

Read More »

Categories

FRAXA Funded Research

Current Research Grants (37)