Finding a Cure for Fragile X
As many of you might have guessed, the 2020 FRAXA Biotech Games had to shift this year to a virtual event. However, the commitment was still there. Throughout the month of September, 39 teams of 4 took time out of their busy schedules to come together every Thursday to connect and show their support for Fragile X research.
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Long-term, but not short-term, treatment with bryostatin-1 — Neurotrope’s lead investigational therapy — arrested such behavioral and cognitive symptoms as hyperactivity, difficulties with daily life activities, and learning and memory deficits in a mouse model of Fragile X syndrome.
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Allos Pharma Inc, a late-stage pharmaceutical company developing therapeutics for neurodevelopmental disorders, has announced the exclusive license rights on IP and documentation for arbaclofen in fragile X syndrome (FXS).
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FRAXA nominated three individuals to join in the program this year. Over the past several weeks, Katie Clapp, Ellen Skala, and Jennifer Frobish participated in the evaluation of research applications submitted to the PRMRP. As consumer reviewers, they were full voting members (along with prominent scientists) at meetings to help determine how the $360 million appropriated by Congress for Fiscal Year 2020 will be spent on PRMRP research.
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Researchers at Stanford University are conducting a clinical research study to learn about behavior & cognition in adults with intellectual & developmental disabilities. Information learned from this research can ultimately contribute to the development of treatment and intervention.
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The Life Span Institute at the University of Kansas currently is seeking one postdoctoral fellow interested in advancing their research skills and progressing towards a career as an independent, externally funded investigator studying autism spectrum (ASD) or related neurodevelopmental disorders (e.g., Fragile X syndrome, Down syndrome).
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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”.
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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.
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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.
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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).
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David Brown, MD, PhD, Ivan Angulo-Herrera, PhD and Anthony Hall of Healx present about the Drug Repurposing Programme for Fragile X syndrome.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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The NIH-sponsored Rare Diseases Clinical Research Network (RDCRN) has created this survey for people living with a rare disease in the US. The goal is to better understand the unique impacts of COVID-19 on the rare disease community.
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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.
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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.
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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.
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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.
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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.
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