Dexmedetomidine Sublingual Film for Autonomic Crises in Familial Dysautonomia
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients with Familial Dysautonomia
This project looks at using a sublingual film medication to help people with familial dysautonomia manage sudden, severe changes in blood pressure and heart rate at home.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | New York University School of Medicine NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11177687 on NIH RePORTER |
What this research studies
Familial dysautonomia (FD) is a rare genetic condition that causes many health challenges, including frequent and severe episodes called hyperadrenergic autonomic crises. These crises involve a sudden spike in blood pressure and heart rate, often with vomiting, and can be dangerous. Current home treatments can have serious side effects like sedation or respiratory problems. This project aims to find a safer and more effective way to manage these crises at home by exploring a sublingual film containing dexmedetomidine, a medication that could help calm the body's overactive stress response during these episodes.
Who could benefit from this research
Good fit: Ideal candidates are patients diagnosed with familial dysautonomia who experience frequent hyperadrenergic autonomic crises.
Not a fit: Patients without familial dysautonomia or those who do not experience hyperadrenergic autonomic crises would not directly benefit from this specific treatment approach.
Why it matters
Potential benefit: If successful, this new treatment could provide a safer and more effective way for patients with familial dysautonomia to manage severe autonomic crises at home, potentially preventing serious complications.
How similar studies have performed: This project proposes a novel approach to treating these crises, indicating that current methods are insufficient and this specific drug delivery for this condition is largely untested.
Where this research is happening
New York, United States
- New York University School of Medicine — New York, United States (Active)
Researchers
- Principal investigator: Gonzalez-Duarte Briseno, Maria Alejandra — New York University School of Medicine
- Study coordinator: Gonzalez-Duarte Briseno, Maria Alejandra
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.