Understanding Morquio A disease and its effects on children
Official title: Non-invasive functional assessment and pathogenesis of Morquio A
Funded at Nemours Children's Hospital, Delaware · NIH-11120885 · Data updated 2026-11-12
This study is looking at how Morquio A disease affects kids' daily lives and health, using non-invasive methods to gather information, so we can find better ways to help manage their symptoms and improve their treatment.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Nemours Children's Hospital, Delaware NIH-funded |
| Lab location | 1 site (Wilmington, United States) |
| Project start | May 1, 2021 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $580,110 |
| NIH project number | 5R01HD102545-05 |
| Project ID | NIH-11120885 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old diagnosed with Morquio A disease.
✕Less likely to help
Patients with other unrelated genetic disorders or those over the age of 11 may not benefit from this research.
What this research studies
This research investigates Morquio A disease, a rare genetic disorder affecting children, caused by a deficiency in a specific enzyme. The study focuses on non-invasive methods to assess the disease's impact on daily activities and overall health. By analyzing medical data from a large patient registry, researchers aim to better understand the progression of the disease and its associated skeletal abnormalities. The findings could lead to improved treatment strategies and better management of symptoms for affected children.
Why it matters
Potential benefit: If successful, this research could lead to enhanced treatment options and improved quality of life for children with Morquio A disease.
How similar studies have gone: Previous research on similar genetic disorders has shown promising results in understanding disease mechanisms and improving patient outcomes.
Where this research is happening
-
Nemours Children's Hospital, Delaware
Wilmington, United States Active
Researchers
- Principal investigator: Tomatsu, Shunji — Nemours Children's Hospital, Delaware
- Study coordinator: Tomatsu, Shunji
Related research
Other NIH-funded projects on the same topics.
-
NCORP pediatric cancer program across Nemours sites
A pediatric cancer network is testing and delivering cancer care and prevention trials to children and young teens across multiple Nemours sites.
-
NEMOURS Move to Improve Blood Pressure Diagnosis in Kids and Teens
A program to help kids and young people with suspected high blood pressure get timely diagnosis by improving access to home-style blood pressure monitoring and sharing easy, local resources.
-
Anticoagulation-free artificial placenta oxygenator for preterm babies
A clinician-scientist is developing a new, safer artificial placenta oxygenator that could help extremely premature infants breathe without needed blood thinners.
-
Improving how people with binge eating disorders learn and use therapeutic skills with digital tools
A study tests digital health tools to help people with binge eating disorders learn and practice cognitive-behavioral skills more effectively.
-
GrowSmart: A kid-friendly AI tool to help primary care teams prevent obesity
A pediatric primary care tool using artificial intelligence helps identify kids at risk for obesity and supports doctors and families in choosing healthy lifestyle steps.
-
Type 1 Together: A teen-focused program to improve CGM use and diabetes health
A teen-centered, peer-supported program tests whether mentor teens and a community health worker can help adolescents with type 1 diabetes wear continuous glucose monitors more consistently and improve health.
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.