New treatments for Niemann-Pick disease type C1
Novel Therapeutic Approaches for NPC Disease
This study is exploring new peptide treatments for Niemann-Pick disease type C1, aiming to help improve cholesterol storage and ease symptoms for people living with this condition.
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-10874651 on NIH RePORTER |
What this research studies
This research focuses on developing innovative peptide therapies for Niemann-Pick disease type C1 (NPC1), a serious genetic disorder that affects the nervous system and other organs. The project aims to understand how these peptides can improve cholesterol storage and alleviate symptoms associated with the disease. Researchers will utilize advanced techniques in biochemistry and molecular biology to study the effects of these treatments on specific biomarkers and overall health outcomes. The goal is to provide a potential new therapy for patients suffering from this condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Niemann-Pick disease type C1, particularly those experiencing neurological and visceral symptoms.
Not a fit: Patients with other types of Niemann-Pick disease or those without a confirmed diagnosis of NPC1 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the first FDA-approved treatment for Niemann-Pick disease type C1, significantly improving patient quality of life.
How similar studies have performed: While there has been limited research on peptide therapies for NPC1, this approach is innovative and aims to fill a significant gap in treatment options for this rare disease.
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.