Neurodevelopmental Drug Development Summit Presentation

FRAXA president and co-founder, Katie Clapp, and and FRAXA Scientific Advisor, Dr. Elizabeth Berry-Kravis, presented at the Neurodevelopmental Drug Development Summit.

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Fragile X Syndrome: In Pursuit of a Cure Webinar

Global webinar “Fragile X Syndrome: In Pursuit of a Cure” on July 22, 2021 commemorated World Fragile X Day. Over 5,000 registered from more than 50 countries.

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Nazim Kourdougli and Carlos Portera-Cailleau

Correcting Sensory Processing in Fragile X Mice by Modulating Kv3.1

FRAXA funded UCLA research on a Kv3.1-targeting drug to ease sensory issues in Fragile X. This work built on Yale-led work now also being pursued by Autifony Therapeutics.

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kaczmarek-Hassar-Brown

Novel Modulators of Potassium Channels to Treat Fragile X

FRAXA-funded Yale research showed disrupted Kv3.1 and Slack potassium channels impair neuronal timing in Fragile X. Published findings support Kv3.1 as a treatment target.

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

FRAXA Research Grants Drive Big Investments in Fragile X

FRAXA is working with hundreds of university labs and more than 30 pharmaceutical companies around the world. Dr. Michael Tranfaglia spends a lot of his time advising and collaborating with industry partners.

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kaczmarek-Hassar-Brown

Newly Discovered Regulatory Pathways in Fragile X

Studies at Yale University and elsewhere show FMRP plays a significant role in regulation of potassium channels. Potassium channel opener drugs could rescue some symptoms of Fragile X.

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Neural Markers of Fragile X: A Powerful New Tool for Clinical Trials

Neural Markers of Fragile X: A Powerful New Tool for Clinical Trials

Once a neural marker is identified for a particular challenge, it can be measured during drug and behavioral therapy trials to see if a child is improving based on objective biological measures.

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FRAXA Funded Research

Current Research Grants (38)