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.
Small-Molecule Inhibitors of Nonsense-Mediated mRNA Decay (NMD) for Fragile X Syndrome
Developing Small-Molecule Inhibitors of Nonsense-Mediated mRNA Decay (NMD) for Clinical Use in Treating Fragile X Syndrome Lynne Maquat, PhD Principal Investigator Hana Cho, PhD FRAXA Postdoctoral Fellow Jordana Schmierer, PhD FRAXA Postdoctoral Fellow University of Rochester Rochester, NY 2024-2025 Grant Funding: $50,000 Summary In February 2021, FRAXA Investigator Dr. Lynne Maquat won the Wolf Prize…
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!
Clinical Study of Non-Invasive EEG for Children Ages 2-7
Dr. Carol Wilkinson, MD PhD at Boston Children’s Hospital recruited children ages 2-7 years with Fragile X syndrome to participate in a study of EEG.
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.
Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome
This Stanford University team assessed combinatorial drug treatments to correct a broad spectrum of deficits observed in Fragile X syndrome. Results published.
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 an advanced new mouse model.
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.
Preclinical Testing of High Fat/Low Carb Diets in Fragile X Mice and Cells
Dr. Cara Westmark’s team will use mice to determine if palatable Atkins-type diets can improve sleep and boost learning skills for those with Fragile X syndrome.
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.