Treatment for abnormal calcium buildup in a rare genetic condition
Official title: Therapy for ectopic calcification in pseudoxanthoma elasticum
Funded at Larix Bioscience, LLC · NIH-10763057 · Data updated 2026-11-12
This study is testing a new oral treatment for people with pseudoxanthoma elasticum (PXE) that aims to help reduce harmful calcification in the body by using a special form of a substance called inorganic pyrophosphate, which could make a positive difference for those living with this rare condition.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Larix Bioscience, LLC NIH-funded |
| Lab location | 1 site (Sunnyvale, United States) |
| Project start | September 21, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $285,766 |
| NIH project number | 1R43AR083332-01 |
| Project ID | NIH-10763057 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with pseudoxanthoma elasticum who experience symptoms related to ectopic calcification.
✕Less likely to help
Patients without a diagnosis of pseudoxanthoma elasticum or those with other unrelated conditions may not benefit from this research.
What this research studies
This research focuses on developing a new oral therapy for patients with pseudoxanthoma elasticum (PXE), a rare genetic disorder that causes abnormal calcification in various tissues, including the eyes and blood vessels. The approach involves using a specially formulated version of inorganic pyrophosphate (PPi) that can be taken orally, which aims to restore normal levels of PPi in the body. By increasing PPi levels, the therapy seeks to inhibit the harmful calcification process associated with PXE. The research will include pharmacokinetic and toxicology studies, followed by evaluations in animal models to assess the effectiveness of the treatment.
Why it matters
Potential benefit: If successful, this research could provide a novel oral treatment option that significantly reduces ectopic calcification in patients with pseudoxanthoma elasticum.
How similar studies have gone: While the approach of using oral PPi for treatment is innovative, similar strategies targeting ectopic calcification have shown promise in preliminary studies.
Where this research is happening
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Larix Bioscience, LLC
Sunnyvale, United States Active
Researchers
- Principal investigator: Mendelsohn, Andrew R — Larix Bioscience, LLC
- Study coordinator: Mendelsohn, Andrew R
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.