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 more![C-subunit Mitochondrial Leak Channel in Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2023/04/Pawel-Licznerski-720x300.jpg)
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.
Read more![Somatosensory Processing as a Therapeutic Target for Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2023/04/Leena-E.-Williams-Andrew-Stanfield-Damien-Wright--720x300.jpg)
Somatosensory 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 more![Celebrating Success at Patrick’s PALS 27th Annual 3-on-3 Basketball Tournament for FRAXA](https://www.fraxa.org/wp-content/uploads/2023/12/Basketball-2-720x300.jpg)
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.
Read more![Antisense Oligonucleotides (ASOs) to restore FMRP in Human Fragile X Cerebral Organoids](https://www.fraxa.org/wp-content/uploads/2023/06/Richter-Shah-Berry-Kravis-Watts-Banbury-Meeting-720x300.jpg)
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.
Read more![FRAXA Research Foundation Partners with Autism BrainNet](https://www.fraxa.org/wp-content/uploads/2023/08/FRAXA-Autism-BrainNet-720x300.png)
FRAXA 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 more![ASOs and Fragile X: Addressing the Most Asked Questions](https://www.fraxa.org/wp-content/uploads/2023/08/Fragile-X-Antisense-Oligonucleotide-therapy-720x300.jpg)
ASOs 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 more![Slack Potassium Channel Inhibitors to Normalize FMR1 Knockout Mice](https://www.fraxa.org/wp-content/uploads/2023/04/Yalan-Zhang-and-Leonard-Kaczmarek-e1683205031523-720x300.jpg)
Slack 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 more![Roles of Postnatal Transient Connectivity in the Development of Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2023/04/Dimitri-Dumontier-Gabrielle-Pouchelon-720x300.jpg)
Roles 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 more![A Tat-Conjugate Approach to Treat Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2023/05/Xiaoqin-Zhan-Raymond-Turner-2-700x300.jpg)
A 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 more![Pioneering Community-Based Drug Development for Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2023/07/Steward-Lab-FRAXA-2-720x300.jpg)
Pioneering Community-Based Drug Development for Fragile X Syndrome
Discover how FRAXA leverages Community-Based Drug Development to create impactful therapies for Fragile X syndrome. Join us as we reshape the future of rare disease treatment.
Read more![The Endocannabinoid System and Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2023/06/Corbin-Laurent-Dionne-720x300.jpg)
The 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 more![Innovative Breakthrough in Fragile X Treatment: The Promise of Antisense Oligonucleotide (ASO) Therapy](https://www.fraxa.org/wp-content/uploads/2023/06/Richter-Shah-Berry-Kravis-Watts-Banbury-Meeting-720x300.jpg)
Innovative Breakthrough in Fragile X Treatment: The Promise of Antisense Oligonucleotide (ASO) Therapy
This changes everything! FRAXA funded research introduces Antisense Oligonucleotide (ASO) Therapy, redefining Fragile X syndrome treatment and understanding.
Read more![Unraveling Fragile X Syndrome: New Insights into FMR1 Gene Reactivation](https://www.fraxa.org/wp-content/uploads/2023/06/Hungoo-Lee-Jeannie-Lee-scaled-e1687181022594-720x300.jpg)
Unraveling Fragile X Syndrome: New Insights into FMR1 Gene Reactivation
Discover groundbreaking methods for reactivating the FMR1 gene in Fragile X syndrome. Dive into the transformational research and the implications of self-healing at a cellular level.
Read more![Allos Pharma Advances Phase 3 Clinical Trial Design for Potential Fragile X Syndrome Treatment, Arbaclofen](https://www.fraxa.org/wp-content/uploads/2020/11/397686_4212565235804_452910278_n-e1605762012898-720x300.jpg)
Allos 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 more![Breakthrough Discoveries in Fragile X Research: Insights from Special Banbury Meeting on Curative Therapies 2023 Fragile X Research Banbury Meeting (1)](https://www.fraxa.org/wp-content/uploads/2023/05/2023-Fragile-X-Research-Banbury-Meeting-1-720x300.jpg)
Breakthrough Discoveries in Fragile X Research: Insights from Special Banbury Meeting on Curative Therapies
Explore the latest breakthroughs in Fragile X research unveiled at the recent Banbury Meeting. Discover novel strategies, from gene therapy to protein replacement, that bring hope for curative therapies.
Read more![Modeling Fragile X Syndrome using Multi-Region Human Brain Organoids](https://www.fraxa.org/wp-content/uploads/2023/04/Yuan-Chen-Tsai-Momoko-Watanabe-720x300.jpg)
Modeling 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 more![SRC Family Kinase Inhibitor as a Potential Treatment for Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2023/04/Smith-Stamenkovic-lab-720x300.jpg)
SRC 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 more![FRAXA Research Foundation Joins COMBINEDBrain Consortium for Fragile X Biomarker Research](https://www.fraxa.org/wp-content/uploads/2023/05/Fragile-X-Biomarker-Research-hero-720x300.png)
FRAXA Research Foundation Joins COMBINEDBrain Consortium for Fragile X Biomarker Research
Help accelerate research on Fragile X syndrome biomarkers by contributing samples to the COMBINEDBrain Consortium’s project. Contact Katie Clapp at FRAXA Research Foundation to learn how you can participate.
Read more![Fragile X Syndrome and Cancer Research: Unexpected Links and Opportunities for Collaboration](https://www.fraxa.org/wp-content/uploads/2023/04/Fragile-X-Syndrome-and-Cancer-scaled-e1680705942845-720x300.jpg)
Fragile X Syndrome and Cancer Research: Unexpected Links and Opportunities for Collaboration
Discover unexpected links between Fragile X Syndrome and cancer. Studies show people with Fragile X have much lower cancer rates. Explore new opportunities for collaboration in this promising research.
Read more![Using Exosomes to Discover Fragile X Biomarkers](https://www.fraxa.org/wp-content/uploads/2023/03/Boussadia_Martire-720x300.jpeg)
Using 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 more![FRAXA Investigator Lynne Maquat Awarded 2023 Gruber Genetics Prize Lynne Maquat](https://www.fraxa.org/wp-content/uploads/2023/03/Lynne-Maquat-720x300.jpg)
FRAXA 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 more![Coming Together for Rare Disease Day 2023](https://www.fraxa.org/wp-content/uploads/2023/02/Rare-Disease-Day-fi-720x300.png)
Coming 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.
Read more![Sex-specific Learning Differences Found in Fragile X Patients, Mouse Model](https://www.fraxa.org/wp-content/uploads/2023/02/Sex-specific-Learning-Differences-Found-in-Fragile-X-Patients-Mouse-Model-700x300.png)
Sex-specific Learning Differences Found in Fragile X Patients, Mouse Model
Girls and women with Fragile X syndrome show different learning impairments relative to boys and men with the disease, a finding that was paralleled in a mouse model of the disease, a study found.
Read more![Targeting Cognitive Function in Fragile X Syndrome](https://www.fraxa.org/wp-content/uploads/2022/11/Steward-Lab-FRAXA-2-720x300.jpg)
Targeting Cognitive Function in Fragile X Syndrome
It has long been assumed that the differences between males and females with Fragile X were simply a matter of degree, with males being more severely affected. But gender differences may be far reaching. This team is working to understand imbalances in how the brain’s neurons transmit signals, with a focus on how differently males and females learn and experience anxiety. They are studying two neuronal pathways which are promising targets for treatment.
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