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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Identifying Cellular and Molecular Signatures in Human Neurons That Distinguish Fragile X Syndrome Patients with Divergent EEG Profiles

Just as Fragile X affects individuals differently, medications do as well. This project aims to bring personalized medicine to Fragile X syndrome.

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Interrogate the Functions of FMRP in Brain Development Using Stem Cells

Dr. Xinyu Zhao of the Waisman Center and Department of Neuroscience at University of Wisconsin-Madison joins us in this seminar to present Interrogate the Functions of FMRP in Brain Development Using Stem Cells.

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Targeting Mitochondria

Targeting Mitochondria in Human Fragile X Syndrome Neurons

Fragile X brain cells have fewer, smaller mitochondria. This team tested mitochondria-boosting drugs that improved symptoms in mice to see if they can help humans.

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Newly discovered aspects of fragile X spur next wave of drugs

Spectrum News – Newly Discovered Aspects of Fragile X Spur Next Wave of Drugs

Many drugs for Fragile X syndrome have failed in large clinical trials, but candidates that target new aspects of the condition may fare better.

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Meng Li, PhD, Xinyu Zhao, PhD, and Anita Bhattacharyya, PhD

FRAXA Research Grants Drive Big Investments in Fragile X

Most people know that FRAXA supports academic research at many institutions such as Harvard University, University of Pennsylvania, Massachusetts Institute of Technology, and Yale University. However, FRAXA is also working with more than 30 pharmaceutical companies around the world. Mike spends a lot of his time advising and collaborating with industry partners.

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Anita Bhattacharyya, PhD

Developing IPS cells to Screen Drugs which can Reactivate the FMR1 Gene

This project developed human stem cell and mouse models to test FMR1 gene reactivation in the brain, advancing future gene therapy strategies for Fragile X.

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FRAXA Funded Research

Current Research Grants (38)