New Ways to Treat Niemann-Pick Type C1 Disease
Novel Therapeutic Approaches for NPC Disease
This project explores new peptide treatments to help manage cholesterol buildup in people with Niemann-Pick disease, type C1.
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-11094087 on NIH RePORTER |
What this research studies
Niemann-Pick disease, type C1 (NPC1), is a serious genetic condition affecting the brain and other organs, for which there is currently no approved treatment. This project aims to develop and understand a new type of peptide therapy designed to reduce the harmful cholesterol buildup seen in NPC1. Researchers will study how these peptides affect specific biological markers and investigate how they work to reduce cholesterol storage. The goal is to find out if these new treatments can improve outcomes and potentially extend life for those with NPC1.
Who could benefit from this research
Good fit: Patients with Niemann-Pick disease, type C1, who currently lack effective treatment options, are the target beneficiaries of this research.
Not a fit: Patients without Niemann-Pick disease, type C1, would not directly benefit from this specific therapeutic approach.
Why it matters
Potential benefit: If successful, this research could lead to the first FDA-approved therapy for Niemann-Pick disease, type C1, offering a new treatment option for patients.
How similar studies have performed: This proposal focuses on developing and understanding a new class of peptides, suggesting a novel and currently untested approach for NPC1.
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.