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.

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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.

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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.

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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.

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Pharmacological Modulation of Nicotinic Signaling

This study tests whether blocking certain nicotine-sensitive receptors in the brain during adolescence can improve attention and cognition in Fragile X.

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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.

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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.

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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.

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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.

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Slack Potassium Channel Inhibitors to Normalize FMR1 Knockout Mice

FRAXA research grant enabled Yale researchers to investigate whether Slack potassium channel inhibitors can normalize behaviors in FMR1 knockout mice.

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The Endocannabinoid System and Fragile X Syndrome

This project will examine how CBD and other drugs targeting the endocannabinoid system affect hyperexcitable Fragile X neurons to identify new treatment strategies.

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SRC Family Kinase Inhibitor as a Potential Treatment for Fragile X Syndrome

This $100,000 FRAXA grant will fuel the Smith lab’s new approach to treating Fragile X syndrome using Saracatinib, originally a cancer drug.

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Using Exosomes to Discover Fragile X Biomarkers

Could a simple blood test show if a Fragile X treatment is working? This team is studying brain-derived exosomes as a new way to track treatment benefits.

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mRNA Therapy for Fragile X Syndrome

Dr. Kathryn Whitehead helped develop the science behind the COVID-19 vaccines. Her team adapted this technology to deliver the Fragile X mRNA to brain cells.

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FRAXA Funded Research

Current Research Grants (38)