Understanding and treating Familial Dysautonomia, a rare nerve disorder
Precision Medicine for the Peripheral Nervous System Disorder Familiar Dysautonomia
['FUNDING_R01'] · UNIVERSITY OF GEORGIA · NIH-10991731
This study is looking at Familial Dysautonomia, a rare condition that affects how the nervous system works, and aims to understand why some people have more severe symptoms than others, so we can find better treatments for those who are affected.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF GEORGIA (nih funded) |
| Locations | 1 site (ATHENS, UNITED STATES) |
| Trial ID | NIH-10991731 on ClinicalTrials.gov |
What this research studies
This research focuses on Familial Dysautonomia, a rare disorder affecting the peripheral nervous system, which leads to severe symptoms like pain insensitivity and blood pressure regulation issues. By using human pluripotent stem cell technology, the researchers aim to recreate the varying severity of symptoms observed in patients to better understand the underlying mechanisms of the disease. The study seeks to identify genetic factors that may influence symptom severity, which could pave the way for targeted therapies. The ultimate goal is to develop precise and effective treatments for patients suffering from this debilitating condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Familial Dysautonomia, particularly those experiencing varying degrees of symptom severity.
Not a fit: Patients with other peripheral nervous system disorders unrelated to Familial Dysautonomia may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the development of targeted therapies that significantly improve the quality of life for patients with Familial Dysautonomia.
How similar studies have performed: While Familial Dysautonomia is a rare condition, similar approaches using stem cell technology have shown promise in understanding and treating other genetic disorders.
Where this research is happening
ATHENS, UNITED STATES
- UNIVERSITY OF GEORGIA — ATHENS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ZELTNER, NADJA — UNIVERSITY OF GEORGIA
- Study coordinator: ZELTNER, NADJA
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.