Finding brain signals to help develop treatments for Rett syndrome
Official title: Development of Translatable Neurophysiological Biomarkers to Accelerate Therapeutic Development in Rett Syndrome
Funded at Children's Hosp of Philadelphia · NIH-11103166 · Data updated 2026-11-12
This study is looking at Rett syndrome to find easy ways to measure how well new treatments are working, using both people and mice, so that we can improve future therapies for those affected by this condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | April 1, 2023 |
| Project end | March 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $701,399 |
| NIH project number | 5R61NS130216-03 |
| Project ID | NIH-11103166 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with Rett syndrome, particularly those under the age of three.
✕Less likely to help
Patients with Rett syndrome who are older than three years may not benefit directly from this specific research.
What this research studies
This research focuses on Rett syndrome, a serious neurodevelopmental disorder caused by mutations in the MECP2 gene. The team aims to develop neurophysiological biomarkers that can indicate treatment responses, which are crucial for advancing therapies. By studying both human patients and mouse models, they will identify non-invasive measures that reflect neurological changes associated with the disease. This approach seeks to enhance the design and execution of clinical trials for new treatments.
Why it matters
Potential benefit: If successful, this research could lead to faster and more effective treatments for children with Rett syndrome.
How similar studies have gone: Previous research has shown promise in using neurophysiological measures as biomarkers in other conditions, suggesting potential success in this novel approach for Rett syndrome.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Marsh, Eric D — Children's Hosp of Philadelphia
- Study coordinator: Marsh, Eric D
Related research
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Neurophysiological biomarkers to speed Rett syndrome therapies
A study to find measurable brain signals that show whether treatments are helping children with Rett syndrome.
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MeCP2 reactivation on the inactive X to treat Rett syndrome
This project explores turning on the silent MECP2 gene on the inactive X chromosome to help people with Rett syndrome feel better.
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Studying how deep brain stimulation helps Rett syndrome in mice
Researchers are testing how deep brain stimulation affects brain cells and memory in a mouse model of Rett syndrome to uncover mechanisms that could guide future therapies.
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Molecular Pathways Behind Rett Syndrome in Humans
A patient-focused look at how changes in the MECP2 gene affect brain function and development in Rett syndrome.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.