Close-up illustration of a neuron and synapses symbolizing brain signaling and BK channel activity in Fragile X syndrome

Opening the Gate: BK Channel Modulation as a Promising Treatment Strategy for Fragile X Syndrome

Fragile X syndrome research is opening the door to BK channel therapies that may calm hyperexcitable brain circuits and improve key symptoms.

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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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Potassium Channel Modulators to Treat Fragile X

FRAXA-backed Yale discoveries tied Fragile X to Kv3.1/Slack channel defects—leading to a partnership with Autifony to develop targeted treatments.

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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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Leonard Kaczmarek, PhD

The Slack Potassium Ion channel is a Therapeutic Target for Fragile X

With $282,000 in funding from FRAXA Research Foundation, Dr. Leonard Kaczmarek and colleagues explored association of Slack channels with the Fragile X protein (FMRP).

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Leonard Kaczmarek, PhD

Electrophysiological, Biochemical and Immunohistochemical Characterization of Kv3.1 in Auditory Brainstem Nuclei in the Fragile X Knockout Mouse

Dr. Leonard Kaczmarek’s Yale lab revealed how Fragile X disrupts potassium channels, impairing auditory processing and fueling sensory overload.

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FRAXA Funded Research

Current Research Grants (38)