Neurophysiological biomarkers to speed Rett syndrome therapies
Official title: Development of Translatable Neurophysiological Biomarkers to Accelerate Therapeutic Development in Rett Syndrome
Funded at Children's Hosp of Philadelphia · NIH-11416178 · Data updated 2026-11-12
A study to find measurable brain signals that show whether treatments are helping children with Rett syndrome.
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, 2028 |
| Funding year | 2026 |
| Award (this year) | $810,952 |
| NIH project number | 4R33NS130216-04 |
| Project ID | NIH-11416178 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates include children with Rett syndrome and suitable mouse models used to validate biomarkers.
✕Less likely to help
Patients who do not participate in studies requiring biomarker measurements or who do not have RTT may not benefit from this biomarker development.
What this research studies
This project aims to identify neurophysiological biomarkers that reflect treatment response in Rett syndrome, using both mouse models and human participants. The team will test candidate signals that are non-invasive and translatable between animals and people, hoping to speed the development of effective therapies. If successful, these biomarkers could guide preclinical and clinical trial design and improve how treatments are assessed in RTT patients.
Why it matters
Potential benefit: If successful, patients with Rett syndrome could benefit from faster, clearer evidence about whether a therapy is working.
How similar studies have gone: Previous work has shown RTT-related neurophysiological differences, but robust, treatment-response biomarkers are still needed.
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
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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.