Cannabinoids as a Treatment for Fragile X Syndrome
This team uses EEG to study sensory hypersensitivity in Fragile X. By testing drugs in mice, they aim to find treatments that calm brain overactivity.
Fundraising Never Tasted So Good
Ice cream for a cause! Lancaster Sweet Shoppe donated proceeds to FRAXA, raising $15,205 in honor of JT. Sweet treats, sweeter impact.
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.
New Fragile X Clinical Trial Announced by Healx
Healx’s AI-driven approach makes finding the right combination therapies more efficient, cost-effective, and rapidly ready for testing at FRAXA-DVI. It was this process that has brought Healx to its recent announcement sharing that it has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for the Phase 2a clinical study of HLX-0201 (sulindac, an FDA-approved drug).
Forbes, “Clinical Trials Expand For AI-Designed Drug To Treat Fragile X Syndrome”
British startup Healx has secured FDA approval for a phase 2a clinical trial of an AI-discovered compound that could help manage the symptoms of the genetic disorder Fragile X syndrome. The start of the trial marks another milestone in the use of artificial intelligence to help find new applications for existing drugs by mining patient records and research databases.
Correcting the Brain’s Emotional Memory Center
FRAXA Investigator Dr. Sumatra Chattarji investigated the synaptic basis of deficient conditioned fear and its reversal in Fragile X syndrome rats. Results published.
Purposeful and FRAXA Partnership Leads to Clinical Trial
AI and FRAXA-DVI identified a drug + supplement combo that reversed all Fragile X symptoms in mice. A clinical trial tested this in adults with Fragile X.
Making Drug Development Efficient Through Community-Based Collaboration
FRAXA’s partnership with Anavex shows how early collaboration between patient advocates and pharma can accelerate drug development for Fragile X and rare diseases.
Meet Matthew!
Meet #FriendofFRAXA Matthew! He is in his final year of school and has always been enthusiastic about his education. He has faced each day with a positive attitude while being friendly and outgoing. Those who meet Matt say he leaves a lasting impression on them. He has been called “The Mayor”, as everyone knows and remembers Matthew where ever goes.
7th Annual 5K Walk for Fragile X in NH Raises Over $3200 For Fragile X Research
The 7th Annual 5K Walk for Fragile X in Derry, NH raised $3,200 for FRAXA. Thank you to everyone who made it a success!
2021 Fragile X Research Grants Funded by FRAXA Research Foundation
Each year, FRAXA funds a diverse portfolio of research. Our FRAXA Fellowships are seed funding for the future, the feedstock for the Fragile X treatment development pathway. While we are looking to promote as many promising new approaches as possible, prominent themes emerge each year, as scientists around the world tackle previously neglected areas.
Inhibiting Nonsense – Mediated mRNA Decay: A Potential Treatment Approach for Fragile X
This team previously discovered runaway nonsense-mediated mRNA decay (NMD) in cells of Fragile X patients. They will now test drugs to reduce NMD.
Exploring Drug Repurposing to Restore Hippocampal Function in FXS Mouse Models
This team found a key mechanism by which FMRP controls brain connections. They’ll test existing drugs that target this pathway to restore learning and memory in Fragile X.
Meet Chloe!
Meet #FriendofFRAXA Chloe! She is a bright girl who loves to make a joke and see people smile! 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.
Contribution of Microglia to the Therapeutic Effects of Metformin and Adiponectin in Fragile X Syndrome
Why are some with Fragile X always hungry or overweight, yet rarely diabetic? This team is studying metabolism and testing treatments like metformin and diet.
20 Years of Advancing Fragile X Research: Progress Toward a Cure
MIT Professor Mark Bear traces the discoveries that give us great optimism of finding effective treatments and ultimately a cure for Fragile X syndrome.
Meet Noah!
Meet #FriendofFRAXA Noah! 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.
Brain Organoids, Moving Fragile X Research Forward
FRAXA-funded scientists at Emory created human brain organoids that reveal Fragile X changes more clearly than mouse models, opening new paths to targeted treatments.
Tetra’s Fragile X Clinical Trial – The Most Successful So Far
Dr. Mark Gurney, CEO of Tetra Therapeutics, discusses how one of the earliest clues to the biology of Fragile X led to the most successful Fragile X clinical trial to date. FRAXA and Tetra began working together after a key FRAXA-funded study caught the attention of Dr. Gurney. Through the FRAXA Drug Validation Initiative, Dr. Patricia Cogram was able to conduct preclinical validation experiments with Tetra’s lead compound in record time, paving the way for clinical trials.
Alternative Splicing in White Blood Cells: A Biomarker for Fragile X Syndrome
This team found 1,600 blood-based Fragile X biomarkers that vary by individual—opening the door to personalized treatment and better ways to measure progress.
Promising Results of Preclinical Study of ANAVEX®2-73
We are excited to share that Anavex Life Sciences announced today that preclinical data of the ANAVEX®2-73 (blarcamesine) study in Fragile X syndrome were published in the peer-reviewed journal, Scientific Reports.
Raising Awareness and Funds for Fragile X Research at Barre3
“A fun and sweaty way to unite the community!” FRAXA thanks Katie Bolin and Barre3’s Katie Heaps for supporting Fragile X awareness.
Fragile X Syndrome and Air Travel
If you wish to participate in a letter writing campaign you can copy the letter below and start sending it the CEOs listed at the bottom of the page.
Meet Jacob!
Meet #FriendofFRAXA Jacob! 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.