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.
A Personal Touch to Supporting Fragile X Research
As the holidays near, take a moment to make a difference. Your year-end gift to FRAXA helps change the future for Fragile X families.
Targeting Adiponectin to Treat Fragile X Syndrome
Boosting adiponectin, a hormone that regulates metabolism, may improve cognition and behavior in Fragile X. Early results suggest it can restore brain plasticity.
Meet Matty & Jackson!
Meet #FriendofFRAXA Matty & 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.
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.
FRAXA Biotech Games, It Can Only Happen in an Open Community
The FRAXA Biotech Games lit up Cambridge Crossing with biotech teams uniting to raise funds and awareness for Fragile X research.
Meet Alfie!
Meet #FriendofFRAXA Alfie! 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.
FRAXA Welcomes Elle to the Team!
FRAXA welcomes Ellen “Elle” Skala as Program Coordinator! She brings grant writing & fundraising experience to support our mission.
Deep Molecular Profiling of Fragile X Mouse and Human Cells
Studying human Fragile X neurons from stem cells revealed key gene changes not seen in mice—showing why some treatments failed and guiding better future therapies.
22 Landmarks Lit up Teal for Fragile X Awareness Day
From one landmark to 20+! On July 22, bridges and buildings across the country lit up teal for Fragile X thanks to FRAXA volunteers.
Targeting Mitochondria in Human Fragile X Syndrome Neurons
Fragile X brain cells have fewer, smaller mitochondria. This team tested mitochondria-boosting drugs that improved symptoms in mice to see if they can help humans.
$19,285 Raised for Fragile X Research at Callum Cup IV
Millburn FC’s 4th Annual Callum Cup raised funds for FRAXA, honoring Callum Murphy, son of goalkeeper Andrew, who has Fragile X.
NH Fragile X Awareness Day Proclaimed at July Jam Fundraiser
The Fragile X July Jam in Derry, NH raised $6,000 for FRAXA! Gov. Sununu’s office also issued a proclamation for Fragile X awareness.
20 Landmarks Will Light Up for Fragile X Awareness Day
20 landmarks in the US & Canada will light up teal on July 22 for International Fragile X Awareness Day, spreading hope and awareness!
Patrick’s PALS 23rd Annual Basketball Tournament Raises $140,000 for Fragile X Research
Patrick’s PALS expanded to 36 teams! The Gray Team topped past champs Kline—winning twice to become Patrick’s PALS XXIII Champions!
Correcting Sensory Processing in Fragile X Mice by Modulating Kv3.1
FRAXA funded UCLA research on a Kv3.1-targeting drug to ease sensory issues in Fragile X. This work built on Yale-led work now also being pursued by Autifony Therapeutics.
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.
Gene Therapy Translational Studies for Fragile X Syndrome
With FRAXA funding, researchers tested AAV gene therapy to restore FMRP in Fragile X mice, measuring safety, effectiveness, and brain activity to inform future trials.
Clinical Trial of Metformin for Fragile X Syndrome
FRAXA-funded open-label trial found that metformin led to increased GABA-mediated cortical inhibition, suggesting metformin modulates core Fragile X pathways.
Developmental Profile of Glutamatergic Synapses in Fragile X
A FRAXA fellowship helped reveal how glutamate receptors at synapses develop differently in Fragile X, offering clues to improve learning and memory.
Reintroducing FMRP via Tat to Reduce Symptoms of Fragile X Syndrome
A FRAXA-funded team found that a shortened FMRP protein, delivered with a Tat “carrier,” restores brain signaling and improves behavior in Fragile X mice.
Enhancing NMDA Receptor Signaling to Treat Fragile X Syndrome
FRAXA-backed work revealed NMDA receptors may hold the key to correcting brain signaling in Fragile X, pointing to new treatment strategies.
FRAXA Biotech Games, the Beginning of Something Great
The first FRAXA Biotech Games united 42 Boston-area teams for friendly competition and fundraising to advance Fragile X research.
Federal Funding for Fragile X Research Faces Snags
Federal support for Fragile X research is declining. NIH and the Department of Defense are reducing investment, making advocacy more important than ever.