FRAXA Biotech Games, the Beginning of Something Great

FRAXA Biotech Games, the Beginning of Something Great

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 having them all back on September 19, 2019, at the Kresge Oval at MIT. Registration and sponsorships for the 2019 FRAXA Biotech Games Cup are available now!

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UMASS CANDI New Techniques to Measure FMR1 mRNA and FMRP

UMASS CANDI New Techniques to Measure FMR1 mRNA and FMRP

CANDI has set a date to wrap up this study by March 2019. Ideally they would like to enroll 5 more individuals with Fragile X syndrome between now and then but if they have at least three more folks who would like to participate, that would be terrific! The upper age limit has been increased from 12 to 22 and they are particularly interested in enrolling girls with Fragile X if possible.

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FRAXA Research Grants Drive Big Investments in Fragile X

FRAXA Research Grants Drive Big Investments in Fragile X

Most people know that FRAXA supports academic research at many institutions such as Harvard University, University of Pennsylvania, Massachusetts Institute of Technology, and Yale University. However, FRAXA is also working with more than 30 pharmaceutical companies around the world. Mike spends a lot of his time advising and collaborating with industry partners.

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Fragile X in the Forefront of International Conference

Fragile X in the Forefront of International Conference

Today the 18th International Fragile X and Related Neurodevelopmental Disorders Workshop kicks off in Quebec, Canada. For the next six days, scientists from around the world will gather at this workshop to focus on recent breakthroughs in our understanding of Fragile X, autism spectrum disorders (ASD), and related neurodevelopmental disorders. This biennial meeting has been instrumental to the discovery of many disease-causing genes and the development of therapeutic strategies for these disorders.

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$75,000 Raised for Fragile X Research by Friends of FRAXA at our Fall Fling

$75,000 Raised for Fragile X Research by Friends of FRAXA at our Fall Fling

On Thursday, September 7 over 150 friends of FRAXA joined us at the Smith Barn at the Peabody Historical Society for the second annual FRAXA Fall Fling. Together we raised over $75,000 for Fragile X research — including a hand-delivered check for $25,000! Families from as far away as Miami, Florida joined us, even with hurricane Irma approaching their home state. Unfortunately, Irma ambushed Weather Channel Storm Tracker and Fragile X parent, Jim Cantore.

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Fulcrum Therapeutics Launched with $55 Million to Reactivate the Fragile X Gene

Fulcrum Therapeutics Launched with $55 Million to Reactivate the Fragile X Gene
A new company has launched that will invest tens of millions in reactivating the Fragile X gene. With $55 million in investment funds, Fulcrum Therapeutics in Cambridge, MA, aim to develop small molecules to control gene expression. These potential new treatments would be based on controlling genetic on- and off-switches of disease genes. Fulcrum will start with two diseases: Fragile X syndrome and a rare form of muscular dystrophy. FRAXA is funding one of the founding scientists, Jeannie Lee, MD, PhD, of Harvard University, and has been working with others on the new Fulcrum team. In fact, Dr. Lee will be our speaker at the FRAXA Fall Fling fundraiser this September 30, in Cambridge, Mass. Together, with your support and with support from new companies like Fulcrum, with support from FRAXA’s Nobel Laureate-laden Scientific Advisory Board and passionate Board of Directors, we are united more than ever in finding a cure for Fragile X. More About Fulcrum http://www.fiercebiotech.com/biotech/third-rock-launches-gene-regulation-startup-55mRead more

FRAXA Grants and Fellowships for Fragile X Research – 2016 Funding Priorities

FRAXA Grants and Fellowships for Fragile X Research – 2016 Funding Priorities
2016 Funding Priorities Start with Clinical Trials While FRAXA Research Foundation’s research goals remain largely unchanged, the landscape in which we operate has changed significantly in the past few years.  Negative results from the major clinical trials of investigational agents have resulted in cessation of development of mGluR5 antagonists for Fragile X syndrome.  There is still much evidence that this drug class could be successful as a Fragile X therapeutic, but we do not see the need for more “proof of principle”-type preclinical research on mGluR5 antagonists. Studies of possible mechanisms of tolerance in Fragile X would be appealing as a topic going forward, as would studies of circuit function in Fragile X, since available evidence suggests some form of circuit-based (rather than synaptic) tolerance in Fragile X mice and humans. Other potential areas of interest would include exploration of combination treatment strategies, both in animal models and in clinicalRead more