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.
Which is the right FMRP for Therapeutic Development of Fragile X Syndrome?
Many forms of FMRP exist in the brain. This project aims to pinpoint which versions of the protein are most critical to restore for effective Fragile X treatments.
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.
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.
One Person Can Make a Difference
Elle Skala launched a $10K birthday fundraiser for FRAXA, promising to shave her head if she reached her goal! What an inspiration!
Ganaxolone Fragile X Clinical Trial Showed Disappointing Results
Ganaxolone, an experimental drug from Marinus Pharmaceuticals which targets GABA receptors, did not show promise for Fragile X syndrome in a clinical trial.
FXS Patients’ Social Deficits are Linked to Social Anxiety, Eye-tracking Study Says
Dr. Craig Erickson and colleagues at the University of Cincinnati used eye-tracking technology to understand sociability in Fragile X syndrome. This study affirms what so many parents, caretakers, and educators suspect: people with fragile X want to be social, and it is anxiety – not lack of interest – which usually hold them back. If anxiety could be reduced, more sociability would likely follow. Dr. Erickson is a Fragile X expert and FRAXA investigator who is currently conducting a Fragile X clinical trial of an investigational new drug.
Fragile X Research has More Runners in the Race
We asked for your help to get more runners in the race to find effective treatments and a cure for Fragile X, and you came through for us. Thank you all so much! FRAXA’s 2018 year-end annual appeal raised $733,017 to fund new Fragile X research!
Screening 2,320 FDA-Approved Drugs for Potential Treatment of Fragile X
FRAXA funded a screen of 2,320 FDA-approved compounds in the Fragile X fly model to identify hits that improve memory and social behavior for advanced testing.