2020 was a year we will never forget. Because of this incredible community and dedicated supporters, we were able to pull through the most difficult year most of us have experienced.
Read moreAuthor: FRAXA Research Foundation
Fragile X Research Funding of $1 Million Offered for 2021
FRAXA plans to fund $1 million for a generous number of Fragile X research grants and fellowships in 2021. Our mission is to find specific treatments and ultimately a cure for Fragile X syndrome. We aim to bring practical treatment into current medical practice as quickly as possible; we prioritize projects that have a clear practical application and the results of which will be shared in a timely fashion.
Read moreIntegrating Human and Mouse Studies in Fragile X Syndrome – an NIH Center Approach
Presentations by:
Craig Erickson – Translational medicine and mechanistic studies of brain neurophysiology in Fragile X Syndrome: A NIH Center Overview
Ernest Pedapati – Network Mechanisms, Biomarkers, and Pharmacology of Fragile X Syndrome in Humans
Devin Binder – Network Mechanisms of Neurophysiology and Behavior in mouse models of Fragile X Syndrome
Kimberly Huber – FMRP Regulation of local and long-range neocortical circuits in the mouse: Links with EEG phenotypes
Developing Arbaclofen for Fragile X – Dr. Mark Bear 1:1 with FRAXA
Seven years ago, arbaclofen (STX209) was pulled from development, disappointing families around the US. Now MIT professor and FRAXA Investigator Dr. Mark Bear has founded Allos Pharma to bring it back. Dr. Bear sat down with FRAXA co-founder Katie Clapp to share the story and next steps.
Read moreTowards Understanding the Role of FMRP in Human Brain Development Using Brain Organoids
Dr. Zhexing Wen and Dr. Peng Jin of the newly funded Fragile X Center of Excellence at Emory University School of Medicine join us in this seminar to present about Understanding the Role of FMRP in Human Brain Development Using Brain Organoids.
Read moreInterrogate the Functions of FMRP in Brain Development Using Stem Cells
Dr. Xinyu Zhao of the Waisman Center and Department of Neuroscience at University of Wisconsin-Madison joins us in this seminar to present Interrogate the Functions of FMRP in Brain Development Using Stem Cells.
Read moreSincere Gratitude for a Successful #GivingTuesday 2020
From the bottom of our hearts, thank you. In a year filled with so much uncertainty, you truly rose to the occasion. From creating Facebook fundraisers to sharing why you give back, each one of you made a difference. With the help of our dedicated supporters and gifts from over 150 amazing donors, FRAXA Research Foundation raised $70,047 on #GivingTuesday.
Read moreAllos Pharma Revives Arbaclofen After 7 Years: New Hope for Fragile X Syndrome
Experience the revival of arbaclofen as Allos Pharma Inc launches a new development program, providing renewed hope for the Fragile X community. Discover the impact of this experimental drug and the determination of those who never gave up.
Read morePositive Results Reported in Phase II Fragile X Clinical Trial of PDE4D Inhibitor Zatolmilast from Tetra Therapeutics
Today, Tetra Therapeutics announces the first unequivocally positive phase 2 clinical trial in Fragile X syndrome, press release below. The results do not depend on carving out a subset of patients or post hoc analysis.
Read moreUse of EEG as a Biomarker for Diagnosis and Outcomes in Neurodevelopmental Disorders
A series webinars focused on current topics in Fragile X research featuring Charles A. Nelson III, PhD, Professor at Harvard Medical School and Carol Wilkinson, MD, PhD, Instructor at Boston Children’s Hospital.
Read moreGenome-wide Screen for FMR1 Reactivation in Human FXS Neural Cells
Drs. Mahmoud Pouladi and Kagistia Utami at the Agency for Science, Technology and Research (A*STAR) in Singapore were awarded a $67,500 research grant from FRAXA Research Foundation and that led to much greater governmental funding to expand this work. Their goal is to reactivate the gene which is silenced in people who have Fragile X syndrome.
Read moreHealx Drug Repurposing Programme for Fragile X Syndrome
David Brown, MD, PhD, Ivan Angulo-Herrera, PhD and Anthony Hall of Healx present about the Drug Repurposing Programme for Fragile X syndrome.
Read moreFRAXA Biotech Games™ 2020 Won by Ginkgo Bioworks
FRAXA Biotech Games brought together the biotech community to network in a friendly setting, form new relationships and potential collaborations while raising money for a great cause! Teams competed for the Cup and for camaraderie, bragging rights, and company pride.
Read moreYale Researcher Dr. Elizabeth Jonas 1:1 with FRAXA
We talk with Dr. Elizabeth Jonas, Professor of Internal Medicine and Neurology at Yale School of Medicine, about her new research suggesting that leaky mitochondria cause some symptoms of Fragile X syndrome. Leaky membranes may also be involved in Parkinson’s Disease and other diseases.
Read moreAuditory System Dysfunction and Drug Tolerance in the Fragile X Mouse
FRAXA Research Foundation has awarded $90,000 over 2019-2021 to principal investigator Dr. Jay Gibson and postdoctoral fellow Dr. Andrew Holley at the University of Texas Southwestern Medical Center. They are investigating circuit mechanisms for auditory system dysfunction and drug tolerance in the Fragile X mouse model.
Read moreMechanisms and Biomarkers of Sensory Hypersensitivity in the fmr1 Knockout Mouse
In this Fragile X research webinar we hear from Devin K. Binder, MD, PhD, Professor, University of California at Riverside Medical School and Khaleel Razak, PhD, Professor, University of California at Riverside as they present about Mechanisms and Biomarkers of Sensory Hypersensitivity in the fmr1 Knockout Mouse.
Read moreA Tribute to Doris Buffett, Philanthropist
We are sad to report that Doris Buffett passed away at the grand age of 92. Doris Buffett donated more than $100 million in her own money to help people who need it. Sister of billionaire Warren Buffett, Doris called FRAXA “The Gold Standard” in grass roots charities.
Read moreFragile X Community Launches #TealTakeover
From a few Zoom Virtual Gatherings an idea was born to do something really special during July to commemorate Fragile X Awareness Day. We would harness the power of the internet to raise awareness with three unique events – the #TealTakeover.
Read moreTalking Step Up for Fragile X
A huge thank you to Talk Fragile X for having FRAXA’s program coordinator Ellen Skala as a guest on your podcast! She got to talk about FRAXA’s upcoming virtual event, Step Up for Fragile X!
Read moreBrain Organoids and Therapeutic Development for Fragile X and Other Rare Diseases
This is the first in a series of webinars focused on current topics in Fragile X research. In this webinar we hear from Alysson R. Muotri, PhD, Professor at University of California San Diego Stem Cell Programand Fabio C. Tucci, PhD, Chief Operating Officer and co-founder at Epigen Biosciences, Inc.
Read moreLandmarks Light Up Fragile X Awareness Day 2020
On Fragile X Awareness Day 2020, 67 landmarks across the US and Canada lit up teal to raise awareness for Fragile X syndrome. Explore the interactive map and celebrate this growing global movement.
Read moreTalk Fragile X Podcast with Dave Bjork
Thank you Talk Fragile X for hosting FRAXA Research Foundation’s very own Dave Bjork as a special guest on your podcast! We greatly appreciate the opportunity to share who we are and what we are doing to advance Fragile X research.
Read moreTrofinetide Clinical Trial Results Published
New Zealand-Based Biotech Neuren Pharmaceuticals Has Published Successful Phase 2 Fragile X Clinical Trial. Trofinetide, given to adolescent and adult males with Fragile X syndrome, was shown to be generally safe and was well-tolerated. It also showed preliminary evidence of efficacy. This trial validated a new design which can be used in future trials.
Read moreScientists Find a New Way to Reverse Symptoms of Fragile X
FRAXA Investigator and MIT Professor Mark Bear and his colleagues have identified a valuable new target for Fragile X therapeutics: GSK3 alpha. Several FRAXA research teams previously identified GSK3 beta as a treatment target for Fragile X. The catch is that, so far, GSK3 beta inhibitors have proven too toxic for regular use. Dr. Bear’s new discovery opens up the possibility of developing more selective compounds with less toxicity and fewer side effects. Interestingly, lithium inhibits both GSK3 versions – alpha and beta.
Read moreMeet Anthony
Meet #FriendofFRAXA Anthony! If you would like to nominate someone as a #FriendofFRAXA, we welcome all who have been touched by Fragile X, including friends, grandparents, siblings, professionals and companions alike to become a #FriendofFRAXA with the goal of putting a face to Fragile X for those who may not know someone directly.
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