Six Potential Treatments Move Forward – 2025 Midyear Impact Report

Explore Our 2025 Midyear Impact Report

Thanks to the incredible support of our global community, the Kabuki Syndrome Foundation is accelerating research at every stage of treatment development. We’ve awarded $560,000 so far this year in the pursuit of delivering treatments faster.

Gifts of all sizes and research participation have made six major research programs possible:

• Two potential treatments are now being tested in Kabuki syndrome mouse models and could be ready for clinical trials as early as next year, with additional funding.
• Three drug repurposing studies are underway to identify existing FDA-approved drugs that could be effective for Kabuki syndrome.
• We’ve also launched early-stage research into a gene regulation approach using dCas9, a tool that could ‘switch on’ the Kabuki gene without altering the DNA itself. 

Dive into our midyear update that highlights these therapeutic studies underway, the growth of our community, and our groundbreaking research that will have a lasting impact. 

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