New treatments for Niemann-Pick disease type C
Novel Therapeutic Approaches for NPC Disease
This study is looking at new peptide treatments for Niemann-Pick disease type C, a genetic condition that affects how the body handles cholesterol, and aims to see if these treatments can help improve health and possibly extend life for those living with this challenging disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Chicago NIH-funded |
| Lab location | 1 site (Chicago, UNITED STATES) |
| Project ID | NIH-11001414 on NIH RePORTER |
What this research studies
This research focuses on developing innovative peptide therapies for Niemann-Pick disease type C (NPC1), a serious genetic disorder that affects cholesterol metabolism and leads to severe neurological and physical complications. The study aims to understand how these peptides can improve cholesterol storage and overall health in NPC1 patients by analyzing specific protein and lipid biomarkers. Researchers will also explore the potential for these treatments to extend lifespan and investigate the underlying mechanisms of action. By utilizing advanced techniques in mass spectrometry and molecular biology, the project seeks to provide new hope for patients suffering from this currently untreatable condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Niemann-Pick disease type C, particularly those experiencing neurological and physical symptoms.
Not a fit: Patients with other forms of Niemann-Pick disease or those without a diagnosis of Niemann-Pick disease type C may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to effective therapies that improve health outcomes and quality of life for patients with Niemann-Pick disease type C.
How similar studies have performed: While there has been limited success with similar approaches, this research is exploring novel peptide therapies that have not yet been tested in the context of Niemann-Pick disease type C.
Where this research is happening
Chicago, UNITED STATES
- University of Illinois at Chicago — Chicago, United States (Active)
Researchers
- Principal investigator: Cologna, Stephanie M — University of Illinois at Chicago
- Study coordinator: Cologna, Stephanie M
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.