Creating a unified database to improve outcomes for children with Metachromatic Leukodystrophy
Official title: Database Harmonization for Creation and Validation of Outcome Assessments and Prediction Tools for Metachromatic Leukodystrophy (MLD)
Funded at Children's Hosp of Philadelphia · NIH-11055648 · Data updated 2026-11-12
This study is working to gather and share information about Metachromatic Leukodystrophy (MLD) in young children to help doctors find out which kids might develop the disease early, so they can get the best care and support as soon as possible.
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 start | June 1, 2025 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $1,049,035 |
| NIH project number | 1R61NS135584-01A1 |
| Project ID | NIH-11055648 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old who are at risk for Metachromatic Leukodystrophy.
✕Less likely to help
Patients who are already diagnosed with advanced stages of MLD may not benefit from this research.
What this research studies
This research focuses on Metachromatic Leukodystrophy (MLD), a rare and progressive neurological disease affecting young children. The project aims to harmonize data from multiple institutions to create a comprehensive database that will help identify which patients are at high risk for early onset MLD. By developing risk prediction models and sensitive assessment tools, the research seeks to enhance early diagnosis and treatment options for affected children. The goal is to ensure that interventions can be administered when children are still minimally symptomatic, potentially improving their quality of life.
Why it matters
Potential benefit: If successful, this research could lead to earlier diagnosis and more effective treatment options for children at risk of MLD.
How similar studies have gone: Previous research has shown success in harmonizing data for rare diseases, indicating that this approach has the potential to yield significant advancements.
Where this research is happening
-
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.