New treatment for severe autonomic crises in patients with familial dysautonomia
Official title: Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients with Familial Dysautonomia
Funded at New York University School of Medicine · NIH-10932210 · Data updated 2026-11-12
This study is looking at a new way to help people with familial dysautonomia manage sudden, severe symptoms using a special film that dissolves under the tongue, which could make it easier and safer for them to handle these episodes 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 start | September 15, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $270,000 |
| NIH project number | 5R01FD007826-02 |
| Project ID | NIH-10932210 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with familial dysautonomia who experience hyperadrenergic autonomic crises.
✕Less likely to help
Patients without familial dysautonomia or those who do not experience autonomic crises may not benefit from this research.
What this research studies
This research investigates a new way to treat hyperadrenergic autonomic crises in patients with familial dysautonomia, a rare genetic disorder. The approach involves using a sublingual film of dexmedetomidine, which may help manage acute episodes that cause severe symptoms like high blood pressure and heart rate, vomiting, and other complications. By testing this treatment, the researchers aim to provide a safer and more effective option for patients to use at home during crises, potentially reducing the need for emergency interventions.
Why it matters
Potential benefit: If successful, this research could provide a safe and effective at-home treatment option for patients experiencing severe autonomic crises.
How similar studies have gone: While this approach is novel for treating autonomic crises in familial dysautonomia, similar methods using dexmedetomidine have shown promise in other contexts.
Where this research is happening
-
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
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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.