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 »

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 »

Innovative Breakthrough in Fragile X Treatment: The Promise of Antisense Oligonucleotide (ASO) Therapy

June 27, 2023

This changes everything! FRAXA funded research introduces Antisense Oligonucleotide (ASO) Therapy, redefining Fragile X syndrome treatment and understanding.

Read More »

10 Year Vision for Fragile X Research – Dr. Elizabeth Berry-Kravis & Dr. Patricia Cogram

December 22, 2022

FRAXA Investigators Dr. Patricia Cogram and Dr. Elizabeth Berry-Kravis reflect on progress that has been made and visualize what they see happening in the next 10 years for people living with Fragile X syndrome.

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 »

FX-Learn Clinical Trial for Children with Fragile X

November 9, 2021

Thirteen centers across the US enrolled young children with Fragile X in a large-scale clinical trial of Novartis AFQ056, an mGluR5 blocker for Fragile X which failed in adult human trials.

Read More »

Purposeful and FRAXA Partnership Leads to Clinical Trial

October 19, 2021

AI and FRAXA-DVI identified a drug + supplement combo that reversed all Fragile X symptoms in mice. A clinical trial tested this in adults with Fragile X.

Read More »

Tetra’s Fragile X Clinical Trial – The Most Successful So Far

September 7, 2021

Dr. Mark Gurney of Tetra Therapeutics, discusses how one of the earliest clues to the biology of Fragile X led to the most successful Fragile X clinical trial to date: a phase 2 trial of a PDE4D inhibitor.

Read More »

Alternative Splicing in White Blood Cells: A Biomarker for Fragile X Syndrome

September 2, 2021

This team found 1,600 blood-based Fragile X biomarkers that vary by individual—opening the door to personalized treatment and better ways to measure progress.

Read More »

2021 FRAXA Awards – Recognizing Perseverance and Dedication

July 22, 2021

In conjunction with World Fragile X Day 2021, FRAXA Research Foundation recognizes its annual award recipients. We are fortunate to partner with these individuals for Fragile X families.

Read More »

Clinical Trials and Cyclic AMP in Fragile X Syndrome: A Life Journey

March 10, 2021

In this Simons Foundation lecture, Elizabeth Berry-Kravis traces 30 years of Fragile X research, from identifying its cause through a series of disappointing clinical trials.

Read More »

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

November 2, 2020

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

Read More »
Tetra Discovery Partners

Tetra Discovery Partners Initiates Phase 2 Trial of BPN14770 in Fragile X Syndrome

July 10, 2018

This 2-Period Crossover Study of BPN14770 is accepting adults males with Fragile X syndrome at Rush University Medical Center in Chicago. Principal Investigator of the study is Elizabeth Berry-Kravis, MD, PhD.
A selective inhibitor of the phosphodiesterase type-4D (PDE4D), BPN14770 has shown the ability to improve the quality of connections between neurons and to improve multiple behavioral outcomes in the Fragile X mouse model.

Read More »
Elizabeth Berry-Kravis, MD, PhD, Fragile X researcher

New Fragile X Clinical Trial for Children Launching in June 2017

October 31, 2016

Elizabeth M. Berry-Kravis, MD, PhD, has launched a large-scale clinical trial to study effects of AFQ056, an mGluR5 blocker, on learning in young children with Fragile X syndrome.

Read More »
Elizabeth Berry-Kravis, MD, PhD, Fragile X researcher

Clinical Trials Outcome Measures

November 22, 2014

There is a critical need for reliable biomarkers and clinical outcome measures for Fragile X syndrome. Treatment trials depend on this.

Read More »
Elizabeth Berry-Kravis, MD, PhD, Fragile X researcher

Pilot Clinical Trial of Lithium in Fragile X Shows Promising Results

November 14, 2010

With $65K from FRAXA, Dr. Berry-Kravis at Rush University ran a pilot lithium trial in 15 Fragile X patients. Results published.

Read More »

Categories

FRAXA Funded Research

Current Research Grants (37)