Department of Defense Medical Research Funding Guided by FRAXA Consumer Reviewers

Department of Defense Medical Research Funding Guided by FRAXA Consumer Reviewers

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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Positive Results Reported in Phase II Fragile X Clinical Trial of PDE4D Inhibitor from Tetra Therapeutics

Positive Results Reported in Phase II Fragile X Clinical Trial of PDE4D Inhibitor 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 analysisAn analysis after seeing the test results, and it can lead to bias, because researchers are able to test the data in any way that produces a favorable result. This trial is the result of many years of rational drug discovery funded by FRAXA. The therapeutic target, PDE4, has been identified by multiple researchers including FRAXA Investigators Tom Jongens, Sean McBride, Elizabeth Berry-Kravis, Nobel laureate and FRAXA scientific advisor Eric Kandel. PDE4 is one of the best validated Fragile X treatment targets and we believe this is a genuinely disease-modifying therapy. (Because PDE4 inhibitors are famous for causing nausea; Tetra's compound targets a subtype of PDE4D, thus largely avoiding GI upset.) Many researchers have expressed doubtsRead more

University of Kansas BRAIN Lab Seeking Postdoctoral Fellow

University of Kansas BRAIN Lab Seeking Postdoctoral Fellow

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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Overcoming the Placebo Effect in Fragile X Clinical Trials

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”.

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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).

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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
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