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.

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Ray Turner, PhD and Xiaoqin Zhan, PhD

Reintroducing FMRP via Tat to Reduce Symptoms of Fragile X Syndrome

A FRAXA-funded team found that a shortened FMRP protein, delivered with a Tat “carrier,” restores brain signaling and improves behavior in Fragile X mice.

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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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kaczmarek-Hassar-Brown

Newly Discovered Regulatory Pathways in Fragile X

Studies at Yale University and elsewhere are showing that FMRP plays a significant role in the regulation of potassium channels. Looking forward, potassium channel opener drugs could rescue some symptoms of Fragile X in humans.

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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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Channelopathies: Altered Ion Channels in Fragile X Syndrome

With a $95,000 grant from FRAXA Research Foundation from 2010-2011, Dr. Daniel Johnston and Dr. Darrin Brager at the University of Texas at Austin investigated alterations in ion channels in Fragile X syndrome. They explored potential therapeutic effects of drugs which open and close these channels. Results published.

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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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