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.
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.
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.
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.
BK Channel Openers: A New Drug for Fragile X Is Ready for Clinical Trials
A promising new BK channel opener, SPG601 from Spinogenix, is entering clinical trials for Fragile X syndrome. Learn about its potential to restore synaptic function and address core symptoms.
Renewed Hope: Navigating Towards a Cure for Fragile X Syndrome
Discover how Dr. Peter Todd’s latest Fragile X Syndrome research offers hope for advanced treatments and a possible cure, marking a new era in FXS therapy.
Inside the FRAXA Drug Validation Initiative: Advancing Fragile X Treatments
FRAXA-DVI is revolutionizing Fragile X syndrome research, providing efficient, comprehensive and objective preclinical testing of potential treatments.
Pharmacologically Activating mGluR7 as a Novel Therapy for Fragile X Syndrome
Join Dr. Tsai and Dr. Kumar on a journey into novel treatments for Fragile X syndrome. Activating mGluR7 could be a game-changer, opening up uncharted therapeutic territory.
Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice
Treating Fragile X might require a combination of drugs. FRAXA-DVI tested two investigational drugs in Fragile X mice. Together ibudilast and gaboxadol rescued a wide array of symptoms.
Unveiling Probiotic Potential in Fragile X Syndrome Clinical Trial
First of its kind in Serbia, this clinical trial explores probiotic intervention as a potential treatment avenue for Fragile X syndrome.
Pharmacological Modulation of Nicotinic Signaling
Nicotine tickles nicotinic acetylcholine receptors which are key to brain functions including learning and memory. The team will see if drugs that dampen these receptors improve cognition in Fragile X.
Astrocyte Contribution to Sensory Hypersensitivity in Fragile X Syndrome
This team studied how faulty calcium signaling in astrocytes contributes to sensory hypersensitivity in Fragile X, aiming to find new astrocyte-targeted treatments.
Reactivating the FMR1 Gene to Reverse Fragile X Syndrome
FRAXA Research Foundation is dedicated to funding breakthrough research, providing $240,000 to reactivate the FMR1 gene to combat Fragile X Syndrome, with the goal of restoring vital protein function and advancing towards a cure.
C-subunit Mitochondrial Leak Channel in Fragile X Syndrome
Explore Yale’s groundbreaking study on mitochondrial leak channels, set to revolutionize Fragile X syndrome treatment. Funded by a $100,000 FRAXA grant.
Somatosensory Processing as a Therapeutic Target for Fragile X Syndrome
FRAXA-funded researchers in Edinburgh assessed a noninvasive touch test that could be used for clinical trials in Fragile X syndrome.
Antisense Oligonucleotides (ASOs) to restore FMRP in Human Fragile X Cerebral Organoids
Explore Dr. Richter’s encouraging results with ASOs for Fragile X syndrome. A $100,000 grant now fuels pivotal studies needed to advance toward ASO therapy.
Slack Potassium Channel Inhibitors to Normalize FMR1 Knockout Mice
Learn how a $100,000 FRAXA research grant supports Yale researchers in using Slack potassium channel inhibitors to treat Fragile X syndrome by normalizing behaviors in FMR1 knockout mice.
Roles of Postnatal Transient Connectivity in the Development of Fragile X Syndrome
This team is studying why people with Fragile X are overly sensitive to sound and light, using advanced imaging to find brain changes and test ways to prevent them.
The Endocannabinoid System and Fragile X Syndrome
Explore groundbreaking research on the potential of Cannabidiol (CBD) in modulating the endocannabinoid system for Fragile X syndrome therapy. Discover how CBD could change the natural course of Fragile X.
Innovative Breakthrough in Fragile X Treatment: The Promise of Antisense Oligonucleotide (ASO) Therapy
This changes everything! FRAXA funded research introduces Antisense Oligonucleotide (ASO) Therapy, redefining Fragile X syndrome treatment and understanding.
Unraveling Fragile X Syndrome: New Insights into FMR1 Gene Reactivation
Discover groundbreaking methods for reactivating the FMR1 gene in Fragile X syndrome. Dive into the transformational research and the implications of self-healing at a cellular level.
Allos Pharma Advances Phase 3 Clinical Trial Design for Potential Fragile X Syndrome Treatment, Arbaclofen
Discover Allos Pharma’s advancements in a pivotal Phase 3 trial for Fragile X syndrome treatment, Arbaclofen. Learn how their FDA-informed trial design might finally bring hope to the Fragile X community.