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 more
Overcoming the Placebo Effect in Fragile X Clinical Trials
In a placebo-controlled clinical trial, some participants are given an experimental medication, while others are given a placebo. Participants do not know whether they are taking medicine or placebo. In theory, this can allow researchers to rule out the placebo effect by comparing outcomes among the two groups. But, per Wexler (2020) “having a strong placebo effect can obscure any real effect of the therapy being investigated”.
Read more
Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest
Zynerba presented clinical trial results for Zygel at a recent neurology conference. Zygel, an experimental cannabidiol (CBD) gel, may reduce behavioral abnormalities in children with Fragile X syndrome who have more severe disease.
Read more
Use 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 more
Centers for Collaborative Research in Fragile X Receive $25 Million Over Next 5 Years
National Institutes of Health (NIH) has announced funding for three Centers for Collaborative Research in Fragile X. The centers will receive $25 million over the next 5 years. Funding for the centers comes from the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Institute of Mental Health (NIMH), and the National Institute of Neurological Disorders and Stroke (NINDS).
Read more
Genome-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 more
Healx 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 more
FRAXA 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 more
Yale 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 more
Auditory 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 more
Mechanisms 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 more
A 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 more
Fragile 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 more
Aripiprazole (Abilify) in the Treatment of People with Fragile X: An Anecdotal Account
The aim of this article is to discuss the use of Abilify (generic name: aripiprazole) as a treatment for people with Fragile X syndrome (FXS). As an “off-label” prescription, Abilify targets behaviors such as irritability, aggression, self-injury and severe tantrums.
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 more
Brain 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 more
Landmarks 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 more
Talk 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 more
Trofinetide 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 more
Scientists 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 more
Meet 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.
Read more
Cholesterol-Dependent Changes in Fragile X Astrocytes
FRAXA Research Foundation has awarded $45,000 to Dr. Maija Castrén, of the University of Helsinki, Finland. Dr. Castren is working with Dr. Iryna Ethell, at the University of California at Riverside, to uncover mechanisms behind beneficial effects of lovastatin and cholesterol-dependent changes seen in the Fragile X brain.
Read more
We’re All In This Together, One Day At A Time.
As many of you know, FRAXA’s mission is to find effective treatment options and ultimately a cure for Fragile X. Right now though, we know that we have to pivot. The Fragile X community has always been the center of our mission and today, there are other ways we can be helpful. We would like to host virtual gatherings that will benefit the Fragile X community and help us all feel connected.
Read more
FRAXA is Thinking About You
We recognize that with the constantly changing COVID-19 situation around the world, this is an unprecedented time for everyone — a time that, for many, is filled with uncertainty. Like many of you, we have children or adults at home with Fragile X syndrome. We truly understand what a great challenge this can be and are here to lend a hand.
Read more
Companies Move to Advance Potential Cognitive Treatment for Fragile X
Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment candidate for disorders marked by cognitive and memory deficits, including Fragile X syndrome and Alzheimer’s disease. The agreement builds on an earlier collaboration between the two companies, and aims to further accelerate BPN14770’s development and potential marketing. It is currently in clinical testing in both Fragile X and Alzheimer’s patients.
Read more