Three More Participants Needed for Fragile X Clinical Trial

Three More Participants Needed for Fragile X Clinical Trial

A Fragile X clinical trial of a new PDE4D allosteric inhibitor from Tetra Therapeutics is nearly complete. Right now there are 3 remaining spots open to males 18-45 years of age with Fragile X syndrome. Dr. Elizabeth Berry-Kravis at the Rush University Medical Center in Chicago is leading this trial. The drug being studied has a unique mechanism of action that might improve cognitive and memory function.  

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National Institutes of Health Releases Fragile X Strategic Plan

National Institutes of Health Releases Fragile X Strategic Plan

FRAXA Program Coordinator, Elle Skala, and long time FRAXA supporter and previous Board Member, Mary Beth Busby, traveled to the National Institutes of Health earlier this week. The timing of this meeting was perfect because the National Institutes of Health (NIH) just released their long-anticipated Strategic Plan for Fragile X Syndrome, FXTAS, and FXPOI. It will guide federal Fragile X research funding for at least the next five years and open the door for continued funding of the Fragile X Research Centers of Excellence.

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Healx Raises $56M to use AI to Find Treatments for Fragile X & Other Rare Diseases

Healx Raises $56M to use AI to Find Treatments for Fragile X & Other Rare Diseases

Healx has secured $56M in new financing to build a clinical-stage portfolio for rare diseases, including treatments for Fragile X syndrome, and to launch a global Rare Treatment Accelerator program. Where the traditional drug discovery model takes more than a decade and can run into the billions of dollars, Healx’s AI-driven approach makes the process faster, more efficient and cost-effective.

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FRAXA Welcomes Elle to the Team!

FRAXA Welcomes Elle to the Team!

Ellen Skala, known as Elle, has joined FRAXA Research Foundation as Program Coordinator. Elle is excited to work on the FRAXA team and we are excited to have her on board! She has experience in grant writing and personal fundraising campaigns. In her new role as program coordinator, Elle will communicate with FRAXA supporters throughout the country, connecting them to the mission and assisting them in fundraising and awareness-raising activities.

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A Day in the Lab with FRAXA Investigator Dr. Tue Banke

A Day in the Lab with FRAXA Investigator Dr. Tue Banke

Recently Laurie Bowler and her 19-year-old son Casey, who has Fragile X syndrome, visited FRAXA research grant recipient Dr. Tue Banke at his University of Washington laboratory. We hope you enjoy Laurie’s wonderful description of their adventure! FRAXA awarded $90,000 to Dr. Banke to study the Developmental Profile of Glutamatergic Synapses in Fragile X.

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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 the upcoming Biotech Games!

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Tetra Announces $40M to Advance BPN14770 for FXS and Alzheimer’s Disease

Tetra Announces $40M to Advance BPN14770 for FXS and Alzheimer’s Disease

Tetra Discovery Partners has signed a multi-part deal that could bring it up to $160 million, plus royalties, from Shionogi & Co, Ltd, a Japanese major research-driven pharmaceutical company. Tetra currently is conducting an investigational Phase 2 study of BPN14770 in adults with Fragile X Syndrome, an indication for which BPN14770 has received Orphan Drug Designation from the US Food and Drug Administration. This clinical trial was made possible by early work with the FRAXA-DVI and over $200,000 from FRAXA.

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Retinoic Acid Signaling is Blocked by Fragile X Mutation

Retinoic Acid Signaling is Blocked by Fragile X Mutation

With a 2013-2014 FRAXA Research Grant, Principal Investigator Marius Wernig, PhD and FRAXA Fellow Samuele Marro, PhD at Stanford University found that the Fragile X mutation impairs homeostatic plasticity in human neurons, by blocking synaptic retinoic acid signaling. Retinoic acid is a metabolite of Vitamin A. The system they have developed could provide a powerful new cellular biomarker for screening many treatment approaches.

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Ringing the Bell at the New York Stock Exchange for Fragile X Awareness

Ringing the Bell at the New York Stock Exchange for Fragile X Awareness

FRAXA Research Foundation was honored to be part of the opening bell ringing ceremony at the New York Stock Exchange (NYSE) on July 5, 2018. We were there to raise awareness for Fragile X along side the National Fragile X Foundation. FRAXA President Katie Clapp and Director of Community Relations Dave Bjork attended with over 30 Fragile X family members and friends, including 4 self-advocates. This was a great opportunity to raise awareness of Fragile X on a big stage at an iconic place. The event was also carried live on television on CNBC, giving FRAXA and Fragile X broad reach around the world.

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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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Impact of the Fragile X Community

Impact of the Fragile X Community

At FRAXA Research Foundation, we are truly grateful for our Fragile X community and thousands of donors. We couldn’t keep moving the ball forward in research without your support. Each year FRAXA invests over $1 million in Fragile X research thanks to your support. Because we supported these three researchers, we were able to secure another $35 million in research aimed at identifying clinical trial outcome measures that will lead to human trials of promising treatments for those affected by Fragile X.

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FRAXA Annual Appeal Raised $760,000

FRAXA Annual Appeal Raised $760,000

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.

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

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

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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Fragile X Awareness Day Origins and a Tribute

Fragile X Awareness Day Origins and a Tribute

July 22 is National Fragile X Awareness Day, but I’ll bet few know the history behind it.

In 2000, before there was such a thing as a Fragile X Advocacy Day, FRAXA Research Foundation and David Busby (husband to Mary Beth, father to two adult sons living with Fragile X, a member of FRAXA’s pioneering leadership team, and a prominent and politically well-connected DC lawyer) were running Fragile X advocacy in Washington, DC.

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