First of its kind in Serbia, this clinical trial explores probiotic intervention as a potential treatment avenue for Fragile X syndrome.
Read moreAuthor: FRAXA Research Foundation
$100,000 Matching Challenge From The Robert & Ardis James Foundation
We are thrilled to announce FRAXA Research Foundation’s most significant and unique matching challenge of the year, thanks to the Robert & Ardis James Foundation. This challenge will help us bring top new talent to Fragile X research.
Read morePharmacological Modulation of Nicotinic Signaling
Nicotine — familiar to any smoker — tickles nicotinic acetylcholine receptors in the brain. These receptors are key to important brain functions including learning and memory. This team will explore whether drugs that dampen these receptors can improve cognitive function in Fragile X.
Read moreAstrocyte Contribution to Sensory Hypersensitivity in Fragile X Syndrome
Most Fragile X research has focused on one type of brain cells: neurons. But mounting evidence point to problems with astrocytes, star-shaped cells which are vitally important to normal brain function. This team is working to understand how astrocytes are involved in Fragile X and develop treatment approaches that targets astrocytes alone.
Read moreYour Support on Giving Tuesday 2023 is Transforming Research
On Giving Tuesday 2023, the generosity of 191 donors raised $333,517 for FRAXA Research Foundation, advancing research and hope for a cure for Fragile X syndrome.
Read moreAn Update from Harmony Biosciences on Giving Tuesday
Harmony Biosciences recently completed its acquisition of Zynerba Pharmaceuticals. I am excited for us to progress their work in pursuit of a treatment option for people living with Fragile X syndrome.
Read moreReactivating the FMR1 Gene to Reverse Fragile X Syndrome
FRAXA Research Foundation is dedicated to funding breakthrough research, providing $240,000 to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function and advancing towards a cure.
Read moreVirtual 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
Read moreC-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.
Read moreSomatosensory Processing as a Therapeutic Target for Fragile X Syndrome
Awarded a FRAXA Research grant, Dr. Andrew Stanfield, Dr. Leena E. Williams, and Dr. Damien Wright are set to explore somatosensory processing (sense of touch) in Fragile X syndrome at the University of Edinburgh. Their aim? A noninvasive touch test that could set the stage for future clinical trials in FXS.
Read moreAntisense 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.
Read moreFRAXA Research Foundation Partners with Autism BrainNet
Discover how FRAXA Research Foundation’s collaboration with Autism BrainNet accelerates Fragile X syndrome research by collecting vital postmortem brain tissue. Dive into the significance of brain studies for deeper insights and potential therapeutic interventions.
Read moreASOs and Fragile X: Addressing the Most Asked Questions
Explore the potential of ASOs in treating Fragile X syndrome & FXTAS. Dive into a comprehensive Q&A addressing key questions and breakthrough findings.
Read moreSupport the FRAXA Dailey Family Campaign
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.
Read moreSlack Potassium Channel Inhibitors to Normalize FMR1 Knockout Mice
Learn how a $100,000 FRAXA research grant supports Yale researchers in using Slack potassium channel inhibitors to treat Fragile X syndrome by normalizing behaviors in FMR1 knockout mice.
Read moreIn Memory of Garret
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.
Read moreRoles of Postnatal Transient Connectivity in the Development of Fragile X Syndrome
A $100,000 FRAXA research grant awarded to Gabrielle Pouchelon, PhD, and Dimitri Dumontier, PhD, at Cold Spring Harbor Laboratory fuels groundbreaking research on Fragile X syndrome. Their work focuses on understanding sensory sensitivities and developing a noninvasive screening method, a potential game-changer for families affected by Fragile X syndrome.
Read moreA Tat-Conjugate Approach to Treat Fragile X Syndrome
Explore Turner Lab’s novel approach to developing definitive treatment for Fragile X syndrome with a Tat-conjugated, truncated FMRP protein. This innovative strategy aims to restore brain circuit function and reduce abnormal behaviors linked to Fragile X by directly addressing the cause of Fragile X: a missing protein.
Read moreThe Endocannabinoid System and Fragile X Syndrome
Explore groundbreaking research on the potential of Cannabidiol (CBD) in modulating the endocannabinoid system for Fragile X syndrome therapy. Discover how CBD could change the natural course of Fragile X.
Read moreAllos Pharma Advances Phase 3 Clinical Trial Design for Potential Fragile X Syndrome Treatment, Arbaclofen
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.
Read moreModeling Fragile X Syndrome using Multi-Region Human Brain Organoids
Discover groundbreaking research at UCI by Dr. Watanabe and Dr. Tsai. Using cutting-edge organoid technology, they’re modeling Fragile X brain function and advancing potential treatment testing.
Read moreSRC Family Kinase Inhibitor as a Potential Treatment for Fragile X Syndrome
Discover the Smith lab’s new approach to treating Fragile X syndrome using Saracatinib, originally a cancer drug. Learn how this $100,000 FRAXA research grant-funded study opens up hopeful avenues.
Read moreUsing Exosomes to Discover Fragile X Biomarkers
How can a blood test give an accurate picture of brain activity? With this grant from FRAXA, Dr. Martire and Dr. Boussadia will try to use unique particles called exosomes – which can travel from brain cells to the blood stream – to evaluate the effects treatments are having on the brain.
Read moreFRAXA Investigator Lynne Maquat Awarded 2023 Gruber Genetics Prize
Dr. Maquat, 2023 Gruber Genetics Prize winner, discovered NMD, a key surveillance system in the body that protects against mistakes in gene expression. Her groundbreaking research has led to new therapies for Fragile X syndrome.
Read moreComing Together for Rare Disease Day 2023
Today, February 28, we mark Rare Disease Day, a day dedicated to raising awareness about rare diseases and highlighting the need for continued research and collaboration. At FRAXA Research Foundation, we are committed to advancing research on Fragile X syndrome, one of the most common rare diseases worldwide.
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