Developing new treatments for capillary malformations in children
Drug Development for Pediatric Capillary Malformation
This study is looking for new medications to help babies with capillary malformations, which are skin issues that can cause health and emotional problems, by understanding how these conditions develop and finding ways to stop them from getting worse.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-10876361 on NIH RePORTER |
What this research studies
This research focuses on capillary malformations (CM), which are vascular anomalies affecting newborns and can lead to significant physical and psychosocial challenges. The project aims to develop pharmacological treatments to prevent the progression of these malformations, which currently have no effective drug therapies. Researchers will create a cell-based assay to study the dysfunction in endothelial cells associated with CM and Sturge-Weber syndrome, which can lead to severe complications. By understanding the underlying mechanisms, the goal is to identify potential drug candidates that could improve patient outcomes.
Who could benefit from this research
Good fit: Ideal candidates for this research are newborns and young children diagnosed with capillary malformations or Sturge-Weber syndrome.
Not a fit: Patients with capillary malformations that are not associated with the specific genetic mutations being studied may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to effective drug treatments for capillary malformations, significantly improving the quality of life for affected patients.
How similar studies have performed: While there is limited research on pharmacological treatments for capillary malformations, the approach of targeting specific genetic mutations has shown promise in other vascular anomalies.
Where this research is happening
Boston, United States
- Boston Children's Hospital — Boston, United States (Active)
Researchers
- Principal investigator: Vivero, Matthew P — Boston Children's Hospital
- Study coordinator: Vivero, Matthew P
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.