FRAXA Research Foundation, Author at Fragile X Research

FRAXA Funded Researchers Present at MA Fragile X Conference

FRAXA Research Foundation Carol Wilkinson, MD, PhD, Clinical Trials, Craig Erickson, MD, News, Recap 04/09/201804/10/2018AZD7325, EEG

On Saturday Boston Children’s Hospital (BCH) hosted a fragile X educational conference, Success Strategies for Individuals and Families Impacted by Fragile X and two of our funded researchers, Dr. Craig Erickson, and Carol Wilkinson, MD, PhD, presented giving an update on their current fragile X clinical trials. Both being funded by FRAXA.

Fragile X Clinical Trial of New PDE4 Inhibitor from Tetra funded by FRAXA

FRAXA Research Foundation 2018 Grants, Clinical, Clinical Trials, Current Research Grants, Elizabeth M. Berry-Kravis, MD, PhD, Rush University Medical Center 04/05/201804/12/2018PDE4, Tetra

With a $200,043 grant from FRAXA Research Foundation in April 2018, Dr. Elizabeth Berry-Kravis will conduct a Phase 2 clinical trial of a new PDE4 inhibitor from Tetra Discovery Partners in adults with fragile X syndrome.

Reintroducing FMRP to Reduce Symptoms of Fragile X Syndrome

FRAXA Research Foundation 2018 Grants, Current Research Grants, Genetic Rescue, Raymond Turner, PhD, University of Calgary, Xiaoqin Zhan, PhD 03/29/201803/30/2018FMRP, Fragile X Research Foundation of Canada, Trans-Activator of Transcription

FRAXA Research Foundation and the Fragile X Research Foundation of Canada have awarded a grant of $100,000 over two years to Dr. Raymond Turner at the University of Calgary in Alberta, Canada. Dr. Turner and postdoctoral fellow Xiaoqin Zhan, PhD are attempting to reactivate a segment of FMRP to reverse symptoms of fragile X in a mouse model of the disease to reduce abnormal behaviors.

Now Recruiting: Clinical Study of EEG for Young Boys at Boston Children’s Hospital

FRAXA Research Foundation Clinical Trials, Recruiting, Research Updates 03/27/201803/29/2018EEG

Dr. Carol Wilkinson, MD PhD, and Dr. Charles Nelson, PhD, at the Labs of Cognitive Neuroscience at Boston Children’s Hospital are recruiting young boys with fragile X syndrome (FXS) to participate in a study investigating how differences in brain activity affect learning, language, and behavior in FXS. If we can determine what distinguishes one brain from another, and if a drug works with a particular neural marker or set of neural markers, this would permit matching drugs based on objective biological markers, a personalized medicine, rather than defaulting to the current method of trial and error.

Neural Markers of Cognitive, Language and Behavioral Deficits in Children with Fragile X

FRAXA Research Foundation 2017 Grants, Biomarkers, Boston Children's Hospital, Carol Wilkinson, MD, PhD, Charles A. Nelson, PhD, Clinical, Current Research Grants, Harvard University, Recruiting 03/16/201804/03/2018

This 2017 grant of $90,000 over two years will enable Dr. Wilkinson to launch a study of EEG in young children with fragile X syndrome at Boston’s Children’s Hospital. She will work with principal investigator, Dr. Charles Nelson, who is a Professor of Pediatrics at Harvard Medical School and a specialist in cognitive neuroscience. Co-funded by the Autism Science Foundation and the Pierce Family Fragile X Foundation.

Fragile X Conference at Boston Children’s Hospital

FRAXA Research Foundation Boston Children's Hospital, Carol Wilkinson, MD, PhD, Craig Erickson, MD, Events 02/12/201802/22/2018

FRAXA and the Pierce Foundation are partners with Boston Children’s Hospital’s Fragile X Program and are pleased to help bring this important fragile X conference to the greater Boston community. Two FRAXA-supported researchers, Dr. Craig Erickson from Cincinnati Children’s Hospital and Dr. Carol Wilkinson from Boston Children’s Hospital will present their current work.

Repurposing Study II: Evaluating Combinations of Drugs to Treat Fragile X

FRAXA Research Foundation 2016 Grants, 2017 Grants, 2018 Grants, Current Research Grants, Drug Repurposing, Healx 02/08/201803/12/2018clinical trial, drug repurposing

FRAXA Research Foundation initially partnered with Healx in 2016 to identify existing drugs with potential to treat fragile X syndrome, using machine learning algorithms and computational biology. The study produced results, and now FRAXA and Healx have launched a new round of studies to evaluate combinations of compounds, including both drugs and natural products.

FX-LEARN – Clinical Trial for Language Learning of AFQ056 in Children

FRAXA Research Foundation Clinical Trials, Elizabeth M. Berry-Kravis, MD, PhD, Recruiting, Rush University Medical Center 02/01/201803/22/2018AFQ056, clinical trial, Novartis

The purpose of this NeuroNEXT study is to find out if the drug AFQ056, made by the pharmaceutical company Novartis, is safe and has beneficial effects on language learning in children who have fragile X syndrome (FXS). The study also aims to find out if a structured language intervention can help children with fragile X syndrome communicate better.

Fragile X Clinical Trial of AZD7325 in Adults

FRAXA Research Foundation 2017 Grants, Biomarkers, Cincinnati Children's Hospital, Clinical, Clinical Trials, Craig Erickson, MD, Current Research Grants 02/01/201804/03/2018AstraZeneca, AZD7325, GABA

With a $51,000 grant from FRAXA Research Foundation, Dr. Craig Erickson will conduct a double-blind, placebo-controlled clinical trial of AZD7325 in adults with fragile X syndrome at Cincinnati Children’s Hospital. The compound being studied is an investigational new drug from AstraZeneca that targets GABA (A) receptors.

FRAXA Annual Appeal Raised $760,000

FRAXA Research Foundation Community, FRAXA Updates 01/16/201802/14/2018

Thanks to your generous support, we surpassed our annual appeal goal of $600,000 and are one step closer to finding effective treatments and, ultimately, a cure for fragile X syndrome. From November 6 to December 31, collectively we raised over $760,000 from over 1,000 donors! This means we will also receive the matching gift of $600,000 from the Sunshine Lady Foundation! We’d like to express our sincere gratitude to all of our donors who generously gave to FRAXA Research Foundation in 2017 and look forward to continuing the promising translational fragile X research momentum in 2018. Our next round of research proposals will be coming in February, and we’re so excited to fund more cutting-edge research. We couldn’t do this without your support. We have an incredible community of families and friends. We are truly grateful for all. We are all in this together.Read more

Novel Modulators of Potassium Channels to Treat Fragile X

FRAXA Research Foundation 2006-2010 Grants, 2011 Grants, 2012 Grants, 2013 Grants, 2014 Grants, 2015 Grants, 2016 Grants, 2017 Grants, Clinical, Current Research Grants, Disease Mechanisms, Leonard Kaczmarek, PhD, Treatment Targets, Yale University 12/25/201704/03/2018auditory neurons, Autifony, hypersensitivity, Kv3.1, Slack

With previous funding from FRAXA, the Yale University team of Leonard Kaczmarek, PhD showed that loss of FMRP leads to an increased Kv3.1 potassium currents and decreased Slack potassium currents in neurons. Both of these changes impair timing of action potentials in auditory neurons (and likely others throughout the brain). The team also found that the firing pattern of neurons in response to repeated stimulation is severely abnormal in fragile X mice. Based on these results, they are collaborating with the UK-based company Autifony to develop and test advanced compounds which may reverse these deficits.

$17,469 Raised by Friends of FRAXA Volunteers at Bruins Game

FRAXA Research Foundation Community, FRAXA Updates, Friends of FRAXA 12/05/201702/21/2018boston bruins, boston bruins foundation

Over 50 #FriendsofFRAXA showed up on the the day after Thanksgiving, November 24, at the Bruins game against the Pittsburgh Penguins to sell 50/50 raffle tickets. Our amazing volunteers sold $34,938 in tickets resulting in $17,469 donated to FRAXA research. This was the highest amount the Bruins sold this year in raffle tickets!

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