Finding a Cure for Fragile X
Zynerba has announced it is now recruiting subjects for a large-scale Phase 3 clinical trial (RECONNECT) of Zygel. Twenty-five sites across the United States, Australia, the UK and Ireland are recruiting children and adolescents with Fragile X syndrome for this randomized, double-blind, placebo-controlled, multinational study, details below.
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Zynerba Pharmaceuticals reported receiving advice from the U.S. Food and Drug Administration (FDA) on the design of an upcoming Phase 3 clinical trial meant to confirm previous trial findings supporting Zygel as a cannabidiol treatment in a specific subset of Fragile X syndrome patients. The new trial, called RECONNECT, is expected to launch before October, and will mainly enroll children and adolescents with a complete (100%) methylation of FMR1, the gene mutated in Fragile X.
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This study will test CBD (cannabidiol) treatment in male and female Fragile X mice to learn how and why it works and whether gender affects responses to CDB treatment. Along with clinical trials, this study will help us to understand and optimize the potential of CBD as a behavior-regulating treatment for Fragile X.
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We are the Bryant Family. Together we are Erin, Joe, Liam, and Lincoln. We want to share with you about our loving son Lincoln.
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When our oldest son, Taylor, was diagnosed at age 2 with Fragile X, the most common inherited cause of mental impairment and autism, brain research suddenly became deeply personal to our family. Please consider making a gift to help accelerate research to find effective treatments and ultimately a cure for Fragile X.
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What can be said about 2020 other than we all want it to be plowed under! It was especially challenging and disruptive for our son/grandson Lucas. His routine is critical to relieving his stress. He mostly blamed us for what he thought was clearly anarchy against his way of living.
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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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On September 20, 2018, FRAXA Research Foundation held the First Annual FRAXA Biotech Games™. The event was a “friendly” competition between greater Boston biotech companies and affiliated industry partners and vendors in a series of fun backyard lawn games. 42 teams of 4 players each played cornhole, KanJam, ladder golf and bucketball. Our goal was to establish an annual event in Cambridge, MA, that would unite the biotech community for an afternoon of fun competition, and raise money for biomedical research. We look forward to the upcoming Biotech Games!
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Administering a cholesterol drug alongside an antibiotic eases atypical behavior and restores the signaling balance in the brains of people with fragile X syndrome.
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With a $90,000 grant from FRAXA Research Foundation over 2018-2019, Drs. Devin Binder, Iryna Ethell, and Patricia Pirbhoy at the University of California at Riverside aim to understand – and reverse – hypersensitivity to sound in Fragile X syndrome.
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New York University scientists make progress developing biomarker signatures and cataloging the types of Fragile X patients who will most likely benefit from new therapies. Take a closer look at your son or daughter with Fragile X syndrome. If you meet another child with Fragile X syndrome, chances are he/she may seem totally different to you, yet everyone is united under a FXS diagnosis. Discovering the biological reasons behind these differences is key to identifying which children will respond to what treatment. But how do you find the ‘prediction formula’? New York University scientists may soon know.
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With a $90,000 grant from the FRAXA Research Foundation from 2013-2014, Dr. Andres Ozaita led a team to test rimonabant’s ability to blockade the CB1 receptor. Blocking CB1 has shown potential to reverse most symptoms of disease in mice bred to mimic Fragile X syndrome.
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Studies have shown that the function of inhibitory networks is disturbed in Fragile X. This abnormality is not well understood but appears to be secondary to abnormalities in metabotropic glutamate and endocannabinoid systems. With a $90,000 grant from FRAXA in 2013-2014, Dr. Molly Huntsman’s team examined how these networks interact and how inhibitory deficits can best be remedied.
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With a $90,000 grant from FRAXA Research Foundation over two years, Drs. Olivier Manzoni and Daniela Neuhofer researched the relationship between Fragile X syndrome and the areas of the brain that are involved in reward processing, regulation of emotional behavior and emotional memory as well as attention, planning and working memory.
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With a $128,500 grant over 2011-2013 from FRAXA Research Foundation, Drs. Bradley Alger and Ai-Hui Tang at the University of Maryland researched endocannabinoid pathways in Fragile X.
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