One of the lesser known signs of Fragile X is unsteady walking. This is also very easy to evaluate in the clinic: no blood tests are required! With a $100,000 grant from FRAXA Research Foundation, this team will develop objective new outcome measures of gait for future treatment trials and also to see if exercise could improve other symptoms of Fragile X.
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$269,774 Raised for Fragile X Research on #GivingTuesday
Thank you very much for being a part of yet another fantastic and historic #GivingTuesday! FRAXA raised a total of $269,774 with our matching contributions!! This is incredible. Our work would be impossible without you.
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A Note of Thanks and Hope on This Giving Tuesday
Why is Fragile X research so important? Every advance in Fragile X brings us all closer to discovering treatments for autism and other brain disorders. We are truly grateful to everyone who has helped FRAXA fund groundbreaking research. Thanks to you, we have hope.
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Clinical Trial Results Reported for Phase 3 CONNECT-FX Study of Zygel™
Results have just been published from Zynerba Pharmaceuticals’s phase 3 clinical trial of Zygel™ in the Journal of Neurodevelopmental Disorders. In this trial, 212 children and adolescents aged 3 to 17 years were given Zygel or placebo for 12 weeks.
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Sigma-1 Receptor as a Therapeutic Target for Fragile X Syndrome
Dr. Pouladi’s team is exploring a treatment of Fragile X via the sigma-1 receptor. Drugs that boost activity at sigma receptors tend to calm down overactive neurons. They are also powerful anti-inflammatory drugs.
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Fragile X Clinical Trial of New PDE4D Inhibitor from Tetra
With a $200,043 grant from FRAXA Research Foundation, Dr. Elizabeth Berry-Kravis completed a successful Phase 2 clinical trial of a PDE4 inhibitor for adult men with Fragile X syndrome. This trial treated 30 males, 18-45 years of age with a new PDE4D allosteric inhibitor from Tetra Discovery Partners using a crossover design, so that everyone got active drug for part of the time and placebo for part of the time.
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$19,600 Raised For Fragile X Research at Callum Cup VI
The Callum Cup is back & as popular as ever! This annual intra-club charity match is the marquee event of the Millburn FC soccer calendar. After 7 years, the Callum Cup matches have raised $94,600 for Fragile X research.
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Versatile Drug Screening Platform for Fragile X Syndrome
Many experts believe that combinations of drugs may be needed to best treat Fragile X syndrome. How can we find the best combinations in the ideal doses? This project — a collaboration between a top university research team and an innovative AI startup both based in Belgium — tackles this challenge.
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Human FMR1 Isoform-Specific Regulation of Translation and Behavior
Fragile X syndrome is caused by lack of one protein, FMRP. But this one protein occurs in different variations. Do the different versions of FMRP have different roles in the brain, and if so, is there one that’s key? If we could replace FMRP to treat Fragile X syndrome, which version would we use?
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Meet Conor!
Meet #FriendofFRAXA Conor! 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.
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Meet Tobias!
Meet #FriendofFRAXA Tobias! 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.
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Functional and Genomic Characterization of Interneurons in the Fmr1-KO Mouse Brain
The brain’s balance is maintained by two types of neurons: those that excite and those that inhibit activity. Like yin and yang, this balance is essential. This team has found fewer than normal inhibitory cells in the brains of Fragile X mice. They are now working to pinpoint this abnormality and find ways to restore the normal balance and function.
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Targeting Serotonin 1A Receptors in Fmr1 Knockout Mice
Dr. Canal has discovered a promising treatment approach for Fragile X syndrome: new compounds which specifically and potently boost serotonin in the brain. The target is the brain’s serotonin 1A receptor.
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FRAXA Volunteer Participates in Peer Reviewed Medical Research Program for the Department of Defense
FRAXA nominated advocate, Jennifer Frobish, recently evaluated research applications submitted to the Peer Reviewed Medical Research Program (PRMRP) of the Congressionally Directed Medical Research Programs (CDMRP).
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Families Come from Multiple States for Flatbread Pizza Fundraiser
The world seems less scary. We were invited to the pizza party where we met Katie and so many other families. We were welcomed like family! It was such a good feeling to not be alone and have people understand your daily struggles and want change, like you, for Fragile X families.
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Patrick’s PALS 3-on-3 Basketball Tournament Returns with a Big Impact
The triumphant return of Patrick’s PALS 3-on-3 Basketball Tournament after a hiatus of three years. With the help of Shady Hill School, the event raised close to $150,000 for FRAXA Research Foundation.
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World Fragile X Day Is Lighting the Way for Fragile X Research
The world shone brightly on July 22 for World Fragile X Day. An impressive 383 locations around the globe lit up to promote awareness and highlight the progress of Fragile X research.
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Why Pharma Companies Take on Fragile X, Explained
Research aimed at finding Fragile X syndrome treatments is exploding. Why are so many pharmaceutical and biotech companies investing in this orphan indication? FRAXA chief scientific officer Dr. Michael Tranfaglia explains the many reasons Fragile X is such a hot topic.
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Charity Golf Tournament on Memorial Day Weekend Helps Fund Fragile X Research
Brothers Pete and Dave Hall hosted their 33rd Annual Charity Golf Tournament on Memorial Day weekend. For the fifteenth consecutive year the money raised from this long-standing event has gone to FRAXA. This year’s total of over $28,000 pushes the 15 year total to over $262,000!
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Transcriptional Signatures Sensitive to Cognition-Improving Pharmacological Treatments in Fragile X Syndrome
The Fragile X field needs biomarkers to accurately measure the effects of potential treatments in both Fragile X mice and in humans. Dr. Ozaita and his team have found molecular features in the brain that can serve as an objective signature for the syndrome. They will use this tool to test cannabidiol and two other drugs in mice.
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Characterization and Modulation of microRNAs in Fragile X Syndrome
Could microRNAs be a new path to treatment of Fragile X syndrome? MicroRNAs are disrupted in Fragile X, and so this team will work to understand what is going wrong and explore ways to correct it with drugs which directly target microRNAs.
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Built Jobsite BBQ Raising Funds for Fragile X Research
Jason, a Built employee, worked with his team to host a BBQ at one of their construction sites to benefit FRAXA Research Foundation. Built, one of Australia’s largest private construction groups, has a reputation for being client focused. Their personal touch extends not only to their clients but to their employees, too, as Jason and Belinda D’Amico experienced personally after their boys, Jaxson and Alex, were diagnosed with Fragile X syndrome.
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Repurposing FDA-Approved Drugs to Treat Major Depressive Disorder in Fragile X Syndrome
Did you know that depression is more common in those with autism and/or Fragile X? Even more disturbing is the discovery that current treatments for depression do not work in Fragile X mice. With this grant, the team will work to develop a rapid screening tool to identify FDA-approved drugs which can treat depression in people with Fragile X syndrome.
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FMR1 Renamed to Fragile X Messenger Ribonucleoprotein 1
The efforts of the European Fragile X Network (EFXN) have led to the renaming of the FMR1 gene to “Fragile X Messenger Ribonucleoprotein 1” gene and the Fragile X protein, FMRP, to “Fragile X Messenger Ribonucleoprotein.” Families around the globe are celebrating the news as a significant step forward for acceptance and the removal of a term that evokes many negative feelings.
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mRNA Therapy for Fragile X Syndrome
Dr. Kathryn Whitehead, Associate Professor at Carnegie Mellon University, helped develop the revolutionary science behind the COVID-19 vaccines. With a $103,000 grant from FRAXA, her team will now adapt this technology to deliver the missing Fragile X protein, to treat people who have Fragile X syndrome.
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