FRAXA Research Foundation

QurAlis and UMass Chan Advance Fragile X Syndrome Treatment using ASOs (Antisense Oligonucleotides)

June 25, 2024

Explore how QurAlis and UMass Chan are revolutionizing Fragile X syndrome treatment using advanced ASO technology, setting new standards in therapeutic development.

Exploring Advances Fragile X Research: Comprehensive Webinar Highlights – May 2024

June 22, 2024

Learn about new FRAXA grants, key clinical trials, and scientific updates that are shaping the future of Fragile X syndrome. Webinar presented by Mike Tranfaglia and Katie Clapp.

Characterization and Modulation of microRNAs in Fragile X Syndrome

June 7, 2024

MicroRNAs are disrupted in Fragile X; the team will work to understand this and explore ways to correct it with drugs which directly target microRNAs.

Celebrate World Fragile X Day: July 22, 2024 – Global Landmarks Light Up

May 29, 2024

Celebrate World Fragile X Day on July 22, 2024 by lighting up landmarks. Join us in honoring Fragile X research and supporting affected families worldwide.

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.

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.

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.

fNIRS to Measure Treatment Response in Young Children with Fragile X

February 13, 2024

The team tested functional near-infrared spectroscopy (fNIRS). fNIRS uses light sources and sensors on the scalp to build a heat map of the brain in action.

Celebrating Teamwork: FRAXA’s 7th Year at Bruins 50/50 Event

February 7, 2024

Celebrating FRAXA’s 7th year at Bruins 50/50 Event: teamwork and community unite for Fragile X research.

Shape Our Conversation: FRAXA Feedback Survey

February 1, 2024

Share your thoughts in our short survey—your insights matter in our 30th year of progress.

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.

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

December 8, 2023

We are thrilled to announce FRAXA’s most significant and unique matching challenge thanks to the Robert & Ardis James Foundation.

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.

Astrocyte Contribution to Sensory Hypersensitivity in Fragile X Syndrome

December 6, 2023

This team studied how faulty calcium signaling in astrocytes contributes to sensory hypersensitivity in Fragile X, aiming to find new astrocyte-targeted treatments.

Your Support on Giving Tuesday 2023 is Transforming Research

November 29, 2023

Thanks to 191 donors, FRAXA raised $333,517 on #GivingTuesday 2023—advancing research and hope for Fragile X families.

An Update from Harmony Biosciences on Giving Tuesday

November 28, 2023

Harmony Biosciences completed its acquisition of Zynerba Pharmaceuticals. This message is from Jeffrey M. Dayno, M.D. Harmony’s President and CEO.

Reactivating the FMR1 Gene to Reverse Fragile X Syndrome

November 7, 2023

This project aims to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function. This work is now funded by a new FRAXA grant.

Virtual Research Q&A with Mike Tranfaglia, MD, and Katie Clapp

November 2, 2023

Please join FRAXA co-founders, Katie Clapp and Dr. Michael Tranfaglia, for a Research Q&A via Zoom on Wednesday, November 15, 2023 at 12:00 pm (noon) ET

C-subunit Mitochondrial Leak Channel in Fragile X Syndrome

September 20, 2023

Explore Yale’s groundbreaking study on mitochondrial leak channels, set to revolutionize Fragile X syndrome treatment. Funded by a $100,000 FRAXA grant.

Somatosensory Processing as a Therapeutic Target for Fragile X Syndrome

September 7, 2023

FRAXA-funded researchers in Edinburgh assessed a noninvasive touch test that could be used for clinical trials in Fragile X syndrome.

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

August 24, 2023

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.