Finding brain signals to help develop treatments for Rett syndrome
Development of Translatable Neurophysiological Biomarkers to Accelerate Therapeutic Development in Rett Syndrome
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 ID | NIH-11103166 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with Rett syndrome, particularly those under the age of three.
Not a fit: Patients with Rett syndrome who are older than three years may not benefit directly from this specific research.
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 performed: 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
Philadelphia, United States
- 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
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 below.