Drug Repurposing Accelerates Progress Towards Fragile X Treatments

While there are over 8,000 rare diseases affecting an estimated 350 million people worldwide, only around 200 of these conditions have effective treatments. Due to the high cost of developing new drugs, rare diseases have historically been less attractive to pharmaceutical companies. Drug repurposing systematically leverages the detailed information available on approved drugs and reduces the time and money needed to deliver safe “new” treatments, but with greater success rates and a potentially more immediate impact on health care.
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Funding Opportunities for Fragile X Research

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FRAXA Wins Award for Drug Repurposing

Cures Within Reach, the leading global nonprofit focused on repurposing research as a fast track to saving patient lives, has awarded FRAXA Research Foundation the 2017 Golan Christie Taglia Patient Impact Philanthropy Award for efforts to find treatments for the rare disease fragile X syndrome.
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Fragile X Research Grants 2017 Recipients

Our lastest Fragile X Research Grants 2017 Recipient is the team of Probal Banerjee, PhD, Alexandra Marsillo, and Tatyana Budylin from College of Staten Island with thier reseach project: Correcting Fragile X-associated Deficits by Targeting Neonatal PKCepsilon Signaling in the Brain.
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Metformin, Diabetes Drug, Potential Fragile X Treatment

"We treated mice with metformin and corrected all the core fragile X deficits. We are optimistic about using metformin in human clinical trials. This is a generic drug with few side effects" says Nahum Sonenberg, PhD, James McGill Professor, Department of Biochemistry, McGill Cancer Center, McGill University.
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Genes and Gems Fundraiser for FRAXA raises $40,000 for Fragile X Research

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TD Bank invites friends of FRAXA to watch the Bruins

Fifty friends of FRAXA enjoyed an amazing night at the TD Garden in Boston on March 30. They gathered to watch the Boston Bruins take on the Dallas Stars. It was a great opportunity for us to unite the greater Boston fragile X community. We were able to convene and thank some of our local families and introduce them to university and biotech scientists who are working to find a cure for fragile X. The night was made available by a generous donation of the Garden View Room by TD Bank.
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Fragile X Nervous (System) Breakdown

"The occurrence and development of events by chance in a happy or beneficial way." That’s how Lynne E. Maquat, PhD, describes the process of how her research extended to fragile X syndrome to better understand it and ultimately find advanced treatments.
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Did tolerance result in Fragile X mGluR5 clinical trial failures?

Although the clinical trials failed to show efficacy in the patient population and Novartis and Roche discontinued their fragile X development programs, Dr. Senter has worked with Mark Bear, PhD to carefully review parent observations. Those caregiver reports suggested tolerance to mGlu5 antagonists antagonists developed quickly, consistent with some preclinical findings in the mouse model.
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Dave Bjork named Director of Development at FRAXA

FRAXA Research Foundation, the world’s only organization solely committed to finding a cure for fragile X syndrome, the leading known inherited cause of intellectual disabilities and autism, has named Dave Bjork to the newly created position of Director of Development.
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Repurposing Available Drugs to Treat Fragile X Syndrome – FRAXA Initiatives

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Fragile X Research Tackles High Anxiety – Peter Vanderklish

Yes, we all know the signs of fragile X anxiety: Ears begin turning red followed by incessant pacing, heavy breathing, stiffening body, flapping, jumping, avoidance or yelling. Sometimes, it’s the more severe screaming, pinching, scratching, biting and general tearing things up or, worse, the nuclear meltdown.
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