Landmarks Light Up Fragile X Awareness Day 2020
On Fragile X Awareness Day 2020, 67 landmarks across the US & Canada lit up teal! Explore the map & celebrate this growing global movement.
Talk Fragile X Podcast with Dave Bjork
Thank you, Talk Fragile X, for featuring FRAXA’s Dave Bjork on your podcast! We’re grateful for the chance to share our mission and research progress.
Trofinetide Clinical Trial Results Published
New Zealand-Based Biotech Neuren Pharmaceuticals Has Published Successful Phase 2 Fragile X Clinical Trial. Trofinetide, given to adolescent and adult males with Fragile X syndrome, was shown to be generally safe and was well-tolerated. It also showed preliminary evidence of efficacy. This trial validated a new design which can be used in future trials.
Scientists Find a New Way to Reverse Symptoms of Fragile X
FRAXA Investigator and MIT Professor Mark Bear and his colleagues have identified a valuable new target for Fragile X therapeutics: GSK3 alpha. Several FRAXA research teams previously identified GSK3 beta as a treatment target for Fragile X. The catch is that, so far, GSK3 beta inhibitors have proven too toxic for regular use. Dr. Bear’s new discovery opens up the possibility of developing more selective compounds with less toxicity and fewer side effects. Interestingly, lithium inhibits both GSK3 versions – alpha and beta.
Meet Anthony
Meet #FriendofFRAXA Anthony! 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.
Cholesterol-Dependent Changes in Fragile X Astrocytes
Astrocytes and cholesterol metabolism are altered in Fragile X. This research uncovers how these changes affect the brain and may reveal new treatment targets like lovastatin.
We’re All In This Together, One Day At A Time.
FRAXA remains dedicated to finding a cure for Fragile X. To support our community, we’re launching virtual gatherings to help us stay connected.
FRAXA is Thinking About You
With COVID-19 changing daily life, we know it’s hard—especially for Fragile X families. FRAXA is here to support you every step of the way.
Ketogenic Diet Eases Symptoms in Fragile X Male Mice
Westmark Lab is studying sleep patterns in Fragile X mice, working with UW-Madison to develop tools that speed EEG data analysis
Meet Jackson!
Meet #FriendofFRAXA Jackson! 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.
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.
fNIRS to Measure Treatment Response in Young Children with Fragile X
The team tested functional near-infrared spectroscopy (fNIRS). fNIRS uses light sources and sensors on the scalp to build a heat map of the brain in action.
Results Reported: Using EEG Responses to Sound for Fragile X Drug Discovery
Jonathan Lovelace, a FRAXA funded Postdoc at UC Riverside, has made some exciting EEG findings over the past few years studying auditory hypersensitivity in mice and therapeutic drug treatments. A big obstacle in FXS research has been establishing reliable, unbiased, and translation relevant biomarkers that can be used to determine the effectiveness of therapies. One of the most important discoveries they have made is the striking similarity in EEG biomarkers between mice and humans.
Should You Participate in a 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.
Meet Will!
Meet #FriendofFRAXA Will! 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.
Thankful is an Understatement
Thanks to our incredible supporters, FRAXA met its $750K goal before 2020—proof that together, we can overcome any challenge!
Boston Bruins Fans and Volunteers raise $18,500 for Fragile X Research
The Bruins Foundation selected FRAXA again for their 50/50 raffle, raising $37K during the Nov 23 game against the Minnesota Wild!
Meet Caleb!
Meet #FriendofFRAXA Caleb! 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.
Jim Cantore Assists on an Absolutely Successful #GivingTuesday
With Jim Cantore’s help and 130+ donors, FRAXA raised $95,500 on #GivingTuesday—funding two year-long Fragile X research grants!
Meet Jed & Kit!
Meet #FriendofFRAXA Jed & Kit! 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.
Genes & Gems Raises $56,000 for Fragile X Research!
Ohio families hosted the unforgettable Genes & Gems event on Nov 16, 2019 at the Renaissance Columbus Hotel to support FRAXA research.
Meet Jack!
Meet #FriendofFRAXA Jack! 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.
Meet Anthony!
Meet #FriendofFRAXA Anthony! 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.
Meet Jagger!
Meet #FriendofFRAXA Jagger! 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.