Fragile X and PDE Inhibitors: A Promising Path Forward for Brain Disorders
Fragile X syndrome research shows PDE inhibitors may improve brain function and behavior, with promise for related neurodevelopmental disorders.
Urgent Action Needed: Help Secure NIH Funding for Fragile X Research
NIH funding delays threaten Fragile X research, putting critical studies and future treatments at risk. Help us urge Congress and the NIH to act now.
Advancing Curative Therapy for Fragile X Syndrome: Turner Lab Secures $1M Grant
Fragile X syndrome research accelerates as the Turner Lab secures $1M to expand FRAXA-funded work on curative therapy targeting the missing FMRP protein.
FRAXA-Funded Research Explores ISRIB as a Potential Treatment for Fragile X
ISRIB for Fragile X syndrome is being studied as a potential treatment to restore brain function and social behavior. Researchers investigate its effects.
Help Direct Millions in Fragile X Research Funding – DOD Seeks Reviewers
The DOD’s Fragile X syndrome research program seeks reviewers to help evaluate funding applications. No science background needed—apply by March 7!
Callum Cup VIII Scores $19,400 for Fragile X Research – A Milestone Event
Callum Cup VIII, Millburn FC’s annual charity match, scored big with $19,400 raised for Fragile X research, bringing total funds to $148,000!
GEXVal and FRAXA Collaborate to Advance Fragile X Research with Phase 2a Trial
GEXVal and FRAXA collaborate to advance Fragile X research with the Phase 2a trial of GXV-001, supported by AMED’s funding program.
19th Annual Fragile X Poker Run Raises $33,000 for Fragile X Research!
The 19th Annual Fragile X Poker Run, hosted by Jimmy, Dana & Mikey Charlton, brought friends & family together in Sparta, NC.
Kicking Off Our 2024 Giving Season With Gratitude and Hope
FRAXA’s 2024 Annual Appeal begins with a $100K gift from the Berg and Schatz families, funding new Fragile X research teams and advancing a cure.
BK Channel Openers: A New Hope for Fragile X Treatment – Insights from Kaerus Bioscience CEO Robert Ring
Kaerus Bioscience’s BK channel openers for Fragile X syndrome are advancing through Phase 1 trials, offering hope for new treatments with FRAXA’s continued support.
Investigating the Role of the Insulin Degrading Enzyme (IDE) in Fragile X Syndrome
This study explores how disrupted insulin signaling affects metabolism and brain function in Fragile X, revealing new treatment targets for both body and mind.
NPR Spotlights Zatolmilast: A Potential Breakthrough for Fragile X Syndrome
NPR spotlights zatolmilast, a promising investigational drug to treat Fragile X syndrome. Families report life-changing improvements in learning and independence.
Altered Physiology of Primary Visual Cortex in Fragile X Syndrome
This team believes inhibitory neurons expressing somatostatin are impaired in Fragile X. They will see if stimulating these neurons has therapeutic potential.
Gene Editing of FMR1 to Correct FXS Phenotypes in Mice
This team is testing cutting-edge gene editing tools to correct Fragile X mutations in the brain. Using a new mouse model, they will see if treatment can restore normal function.
Fragile X Unplugged: Establishing Mobile EEG as the Next Frontier
A $100,000 FRAXA grant to Cincinnati Children’s Hospital is simplifying and testing EEG technology for home use, improving clinical trial accessibility and efficiency.
Marvel Biosciences Partners with FRAXA to Test MB204 for Fragile X Syndrome
Marvel Biosciences and FRAXA Research Foundation are collaborating to test MB204, a promising new treatment for Fragile X which targets adenosine receptors.
World Fragile X Day 2024: A Global Celebration of Awareness and Research Progress
Recap of World Fragile X Day 2024, a global event spearheaded by FRAXA. Explore beautiful photos of landmark illuminations around the world.
Identification of the Proteome of Active and Silenced FMR1 Alleles in Human Stem Cells
This project aims to uncover which proteins keep the Fragile X gene silenced. By identifying them, the team hopes to find new ways to switch the FMR1 gene back on.
Understanding Anxiety in Fragile X: Insights from Dr. Carrie Buchanan’s Webinar
Dr. Carrie Buchanan shares strategies to manage anxiety in individuals with Fragile X syndrome, including non-pharmacological treatments and medication options.
To Interrogate the Developmental Timing for Treating Fragile X Syndrome
Are there critical periods in Fragile X syndrome? Will treatment work in adults as well as in children? This team aims to answer these questions.
Hole-in-One for Fragile X Research: Highlights from the 37th Annual Charity Golf Tournament
The Hall family’s golf tournament raised $35,000 for Fragile X research—bringing their total contributions to over $327,000 for FRAXA!
Patrick’s PALS 28th Annual Basketball Tournament for FRAXA Breaks Record
Recap of Patrick’s PALS 28: a successful 3-on-3 basketball fundraiser in Boston, raising $170,000+ for FRAXA Research Foundation.
QurAlis and UMass Chan Advance Fragile X Syndrome Treatment using ASOs (Antisense Oligonucleotides)
Explore how QurAlis and UMass Chan are revolutionizing Fragile X syndrome treatment using advanced ASO technology, setting new standards in therapeutic development.
Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration
FRAXA pharma partner Neurolixis has filed patents for treatment of Fragile X with a highly selective new drug, NLX-101, which targets the serotonin receptor 5-HT1A.