Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration

FRAXA pharma partner Neurolixis has filed patents for treatment of Fragile X with a highly selective new drug, NLX-101, which targets the serotonin receptor 5-HT1A.

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

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

A promising new BK channel opener, SPG601 from Spinogenix, is entering clinical trials for Fragile X syndrome. Learn about its potential to restore synaptic function and address core symptoms.

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

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.

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Harmony Biosciences Phase 3 Clinical Trial (RECONNECT) with At-Home Option

Harmony Biosciences’ RECONNECT Phase 3 trial of ZYN002 offered an at-home participation option. Unfortunately the trial reported negative results.

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Renewed Hope: Navigating Towards a Cure for Fragile X Syndrome

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.

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Inside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments

FRAXA-DVI is revolutionizing Fragile X syndrome research, providing efficient, comprehensive and objective preclinical testing of potential treatments.

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Pharmacologically Activating mGluR7 as a Novel Therapy for Fragile X Syndrome

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.

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Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice

Treating Fragile X might require a combination of drugs. FRAXA-DVI tested ibudilast and gaboxadol in Fragile X mice. Together they rescued a wide array of symptoms.

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

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

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Shape Our Conversation: FRAXA Feedback Survey

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

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Callum Cup 2023 Group

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

The Callum Cup in Millburn, NJ raised $26K for Fragile X research—a charity soccer match celebrating community and compassion!

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Unveiling Probiotic Potential in Fragile X Syndrome Clinical Trial

First of its kind in Serbia, this clinical trial explores probiotic intervention as a potential treatment avenue for Fragile X syndrome.

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

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

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Pharmacological Modulation of Nicotinic Signaling

This study tests whether blocking certain nicotine-sensitive receptors in the brain during adolescence can improve attention and cognition in Fragile X.

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Astrocyte Contribution to Sensory Hypersensitivity in Fragile X Syndrome

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

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Your Support on Giving Tuesday 2023 is Transforming Research

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

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An Update from Harmony Biosciences on Giving Tuesday

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

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Reactivating the FMR1 Gene to Reverse Fragile X Syndrome

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.

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

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

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C-subunit Mitochondrial Leak Channel in Fragile X Syndrome

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

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Somatosensory Processing as a Therapeutic Target for Fragile X Syndrome

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

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Celebrating Success at Patrick’s PALS 27th Annual 3-on-3 Basketball Tournament for FRAXA

Recap of Patrick’s PALS 27: a successful 3-on-3 basketball fundraiser at Bentley University, raising $150,000+ for FRAXA Research Foundation.

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

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.

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FRAXA Funded Research

Current Research Grants (38)