Investigating treatments for Aicardi Goutières Syndrome in children
Clinical measures in Aicardi Goutieres Syndrome
This study is looking at how well a medication called baricitinib can help children with Aicardi Goutières Syndrome feel better and improve their daily lives, while also learning more about the different ways this condition affects kids.
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-10689193 on NIH RePORTER |
What this research studies
This research focuses on Aicardi Goutières Syndrome (AGS), a genetic disorder that leads to early neurological disabilities in children. The study aims to evaluate the effectiveness of anti-inflammatory medications, specifically baricitinib, in reducing symptoms and improving the quality of life for affected children. Researchers will assess the neurological progression and response to treatment using various clinical measures and patient-reported outcomes. By understanding the different phenotypes of AGS, the research seeks to develop better assessment tools and therapeutic strategies for this condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with Aicardi Goutières Syndrome, particularly those experiencing neurological impairments.
Not a fit: Patients with other neurological disorders or those not diagnosed with Aicardi Goutières Syndrome may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment options and better management of symptoms for children with Aicardi Goutières Syndrome.
How similar studies have performed: Previous research has shown promising results with similar anti-inflammatory approaches in other neurological conditions, suggesting potential for success in this area.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Adang, Laura Ann — Children's Hosp of Philadelphia
- Study coordinator: Adang, Laura Ann
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.