Centers for Collaborative Research in Fragile X Receive $25 Million Over Next 5 Years

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

Auditory System Dysfunction and Drug Tolerance in the Fragile X Mouse

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

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

Aripiprazole (Abilify) in the Treatment of People with Fragile X: An Anecdotal Account

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 more

Brain Organoids and Therapeutic Development for Fragile X and Other Rare Diseases

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

Landmarks Light Up Fragile X Awareness Day 2020

This year, on Fragile X Awareness Day, a growing number of buildings, bridges, and monuments — including Niagara Falls — will be illuminated in teal to spread the word about Fragile X and celebrate our community. Although we may not be able to gather in groups this year, we still encourage you to briefly visit or drive by lighting displays and share your experience with us.

Read more

FRAXA Biotech Games™ Reimagined as Online Event for 2020

FRAXA Biotech Games™ Reimagined as Online Event for 2020

FRAXA Biotech Games™, a gathering of Boston area biotech companies to network in a friendly setting, and form new relationships and potential collaborations while raising money for Fragile X research, is adapting its 2020 event to maintain its commitment to Fragile X and autism research while prioritizing the health and safety of its contestants and volunteers. The organization’s traditional annual event will be transformed into an online interactive experience September 9-11, 2020.

Read more

Trofinetide Clinical Trial Results Published

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

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.

Bear lab (Bear 3rd from left, McCamphill on right)Read more

Meet Anthony

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

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.

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

Companies Move to Advance Potential Cognitive Treatment for Fragile X

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

Ketogenic Diet Eases Symptoms in Fragile X Male Mice

Ketogenic Diet Eases Symptoms in Fragile X Male Mice

The Westmark laboratory continues to study sleep and rest-activity cycles in Fragile X mice as a potential outcome measure that correlates between preclinical and clinical research. The analysis of sleep EEG in the mice has proven more labor intensive than they anticipated, but the team is collaborating with Dr. Rama Maganti’s laboratory at UW-Madison on the development of computer scrips to speed up the analysis.

Fragile X Researcher, Cara Westmark, PhDRead more

Drug Repurposing for Rare Disease and the Future of Health – The Genetics Podcast

Drug Repurposing for Rare Disease and the Future of Health – The Genetics Podcast

In this double-bill episode of The Genetics Podcast, Dr. Patrick Short talks to two key rare disease researchers in the field: Dr. Bruce Bloom, CCO of Healx, and Dr. Mike Tranfaglia, CSO of FRAXA. Both draw on their wide-ranging personal and professional experiences to discuss the successes and opportunities of drug repurposing, the power of using machine learning, and the work they’ve been doing to aid in finding effective treatments for Fragile X.

Read more
Copy link