BK Channel Openers: A New Hope for Fragile X Treatment – Insights from Kaerus Bioscience CEO Robert Ring

Kaerus Bioscience’s BK channel openers for Fragile X syndrome are advancing through Phase 1 trials, offering hope for new treatments with FRAXA’s continued support.

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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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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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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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FRAXADev – Developing BK Channel Openers for Fragile X Syndrome

A number of people have asked us about FRAXADev, a new project starting in France; this is a nonprofit initiative which seeks to develop a new kind of drug for Fragile X. The drugs they are interested in testing in Fragile X clinical trials were developed by Bristol-Myers Squibb many years ago, and are now off patent. This class of drugs opens a potassium channel in the membrane of neurons, which helps to decrease neuronal excitability.

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Fragile X Treatment: New Research Directions

In the wake of negative results from several high-profile clinical trials in Fragile X, we find ourselves questioning many of our previous assumptions about the nature of this disorder. After all, understanding the basic pathology of disease is critical to development of new treatments — this is true across the board, in all branches of medicine.

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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

Ion channel defects (“channelopathies”) in Fragile X disrupt neuron firing and network balance. This study maps these channel changes to guide targeted treatments.

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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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FRAXA Funded Research

Current Research Grants (45)