FRAXA-Funded Research Explores ISRIB as a Potential Treatment for Fragile X

February 26, 2025

ISRIB for Fragile X syndrome is being studied as a potential treatment to restore brain function and social behavior. Researchers investigate its effects.

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Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

March 9, 2021

This Stanford University team assessed combinatorial drug treatments to correct a broad spectrum of deficits observed in Fragile X syndrome. Results published.

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Synaptic Characterization of Human Fragile X Neurons

September 12, 2014

Stanford scientists used human stem-cell–derived neurons to show that retinoic acid signaling is blocked by Fragile X, revealing a new pathway to target for treatment.

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Composition and Localization of Dendritic mRNAs in Fragile X Syndrome

July 9, 2009

With an $80,000 FRAXA grant, Drs. Smith and Wang developed a system to visualize proteins and mRNAs in Fragile X neurons, revealing how regulation goes awry.

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FRAXA Funded Research

Current Research Grants (42)