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.

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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.

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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.

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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.

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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

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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.

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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.

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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.

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FRAXA Research Foundation Partners with Autism BrainNet

August 17, 2023

FRAXA’s collaboration with Autism BrainNet can accelerate Fragile X syndrome research by collecting vital postmortem brain tissue.

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ASOs and Fragile X: Addressing the Most Asked Questions

August 16, 2023

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

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Support the FRAXA Dailey Family Campaign

August 15, 2023

Join the Dailey Family in their journey with Fragile X syndrome. Meet Kristin, a renowned marathon runner, Sean, with strong Boston roots, and their four children, including Declan with Fragile X.

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Slack Potassium Channel Inhibitors to Normalize FMR1 Knockout Mice

August 14, 2023

FRAXA research grant enabled Yale researchers to investigate whether Slack potassium channel inhibitors can normalize behaviors in FMR1 knockout mice.

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In Memory of Garret

August 11, 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.

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The Endocannabinoid System and Fragile X Syndrome

July 6, 2023

This project will examine how CBD and other drugs targeting the endocannabinoid system affect hyperexcitable Fragile X neurons to identify new treatment strategies.

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

May 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.

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Modeling Fragile X Syndrome using Multi-Region Human Brain Organoids

May 16, 2023

The team is developing new, more accurate Fragile X brain organoids to help researchers study neural circuit problems and accelerate testing of future treatments.

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SRC Family Kinase Inhibitor as a Potential Treatment for Fragile X Syndrome

May 7, 2023

This $100,000 FRAXA grant will fuel the Smith lab’s new approach to treating Fragile X syndrome using Saracatinib, originally a cancer drug.

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Using Exosomes to Discover Fragile X Biomarkers

March 21, 2023

Could a simple blood test show if a Fragile X treatment is working? This team is studying brain-derived exosomes as a new way to track treatment benefits.

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Lynne Maquat

FRAXA Investigator Lynne Maquat Awarded 2023 Gruber Genetics Prize

March 8, 2023

Dr. Maquat discovered NMD, a key surveillance system in the body that protects against mistakes in gene expression. With funding from FRAXA she is tackling Fragile X syndrome.

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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.

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Targeting Cognitive Function in Fragile X Syndrome

February 14, 2023

Why do males and females experience Fragile X differently? This team is studying brain signaling pathways to uncover sex-based differences and guide treatments.

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Validating Novel Inhibitors of ERK Signalling to Treat Fragile X Syndrome

February 13, 2023

This team is testing ERK inhibitors — drugs that may calm overactive brain signaling in Fragile X. Early results in mice show strong benefits with minimal side effects.

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A Look Back at 2022 and Ahead to 2023 Research Prospects

January 18, 2023

FRAXA supporters met the $100K Giving Tuesday challenge—raising $269,744! Your actions truly spoke louder than words. Thank you!

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Targeting Serotonin 1a Receptors to Reverse Neurobehavioral Phenotypes

January 14, 2023

Neurolixis’ new drug targets serotonin 1A receptors, showing promise in preclinical studies for Fragile X syndrome, funded by a FRAXA grant for future clinical trials.

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FRAXA Funded Research

Current Research Grants (38)