Clinical Trials and Cyclic AMP in Fragile X Syndrome: A Life Journey

Clinical Trials and Cyclic AMP in Fragile X Syndrome: A Life Journey

In November 2020, a phase II clinical trial reported extremely successful results. This clinical trial of a PDE4D inhibitor from Tetra Pharmaceuticals was conducted by Dr. Elizabeth Berry-Kravis at Rush University Medical Center and funded by FRAXA Research Foundation. In this Simons Foundation lecture, Elizabeth Berry-Kravis traces 30 years of Fragile X research, from identifying its cause, through finding dozens of treatment targets, through a series of disappointing clinical trials.

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Fragile X Research Funding of $1 Million Offered for 2021

Fragile X Research Funding of $1 Million Offered for 2021

FRAXA plans to fund $1 million for a generous number of Fragile X research grants and fellowships in 2021. Our mission is to find specific treatments and ultimately a cure for Fragile X syndrome. We aim to bring practical treatment into current medical practice as quickly as possible; we prioritize projects that have a clear practical application and the results of which will be shared in a timely fashion.

FRAXA Grant ApplicationsRead more

Centers for Collaborative Research in Fragile X Receive $25 Million Over Next 5 Years

Centers for Collaborative Research in Fragile X Receive $25 Million Over Next 5 Years

National Institutes of Health (NIH) has announced funding for three Centers for Collaborative Research in Fragile X. The centers will receive $25 million over the next 5 years. Funding for the centers comes from the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Institute of Mental Health (NIMH), and the National Institute of Neurological Disorders and Stroke (NINDS).

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Landmarks Light Up Fragile X Awareness Day 2020

Landmarks Light Up Fragile X Awareness Day 2020

This year, on Fragile X Awareness Day, a growing number of buildings, bridges, and monuments — including Niagara Falls — will be illuminated in teal to spread the word about Fragile X and celebrate our community. Although we may not be able to gather in groups this year, we still encourage you to briefly visit or drive by lighting displays and share your experience with us.

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We’re All In This Together, One Day At A Time.

We’re All In This Together, One Day At A Time.

As many of you know, FRAXA’s mission is to find effective treatment options and ultimately a cure for Fragile X. Right now though, we know that we have to pivot. The Fragile X community has always been the center of our mission and today, there are other ways we can be helpful. We would like to host virtual gatherings that will benefit the Fragile X community and help us all feel connected.

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Companies Move to Advance Potential Cognitive Treatment for Fragile X

Companies Move to Advance Potential Cognitive Treatment for Fragile X

Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment candidate for disorders marked by cognitive and memory deficits, including Fragile X syndrome and Alzheimer’s disease. The agreement builds on an earlier collaboration between the two companies, and aims to further accelerate BPN14770’s development and potential marketing. It is currently in clinical testing in both Fragile X and Alzheimer’s patients.

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Drug Repurposing for Rare Disease and the Future of Health – The Genetics Podcast

Drug Repurposing for Rare Disease and the Future of Health – The Genetics Podcast

In this double-bill episode of The Genetics Podcast, Dr. Patrick Short talks to two key rare disease researchers in the field: Dr. Bruce Bloom, CCO of Healx, and Dr. Mike Tranfaglia, CSO of FRAXA. Both draw on their wide-ranging personal and professional experiences to discuss the successes and opportunities of drug repurposing, the power of using machine learning, and the work they’ve been doing to aid in finding effective treatments for Fragile X.

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New Partners Aim to Accelerate the Discovery and Repurposing of Medicines for Rare Neurological Diseases

New Partners Aim to Accelerate the Discovery and Repurposing of Medicines for Rare Neurological Diseases

First Healx secured $56M in new financing to launch a global Rare Treatment Accelerator program to tackle Fragile X syndrome and 39 other rare diseases. Now they have built a partnership with Boehringer Ingelheim worth millions. It all started with a small FRAXA grant to Healx to repurpose available drugs for Fragile X.

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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.

Elle Skala, Tracy King, Christie Rogers, Mary Beth BusbyRead more

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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