Big News for the TBRS Community: $75,000 Grant to Advance Gene Therapy Research

We are thrilled to share exciting news with our TBRS families and everyone who supports this community.

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The Tatton Brown Rahman Syndrome Community has received a $75,000 grant from the RTW Foundation to support gene therapy research for TBRS at Washington University in St. Louis, led by Dr. Harrison Gabel.

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This is a meaningful step forward in our long-term goal of developing treatments that address the underlying causes of TBRS.

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What does this grant support?

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This funding will support early-stage gene therapy research in Dr. Gabel’s lab. Gene therapy research focuses on finding ways to correct or compensate for changes in a gene that cause a condition. In TBRS, the condition is caused by changes in the DNMT3A gene.

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While this work is still in the research phase and not a treatment available today, it is an essential part of building the foundation for future therapies. Research like this helps scientists understand what approaches may be possible, what is safe, and what is most promising to pursue next.

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Why Dr. Gabel and Washington University?

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Dr. Harrison Gabel is a leading neuroscientist at Washington University in St. Louis and a valued member of the TBRS Community Medical and Scientific Advisory Committee. He has been deeply engaged with the TBRS Community and understands both the science and the lived experiences of individuals and families affected by TBRS.

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Funding work in Dr. Gabel’s lab means investing in research that is guided by real patient needs and grounded in deep expertise.

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About the RTW Foundation

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The RTW Foundation is a longtime partner to the TBRS Community. In addition to providing this $75,000 research grant, RTW also offers advisory services to our organization at no cost, helping us strengthen our scientific strategy and accelerate progress for TBRS and other ultra-rare diseases.

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RTW’s commitment goes beyond funding. They actively support ultra-rare disease research and help patient communities like ours navigate complex scientific pathways with care and integrity.

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Why this matters to families

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For many TBRS families, the idea of gene therapy represents hope, not just for symptom management, but for addressing TBRS at its root.

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We are deeply grateful to the RTW Foundation for their trust in our community and to Dr. Gabel for his dedication to advancing TBRS research. This progress is possible because of the collective strength of families, researchers, advisors, and supporters who believe in a better future for people with TBRS.

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