Medical records reveal clues to rare genetic disease

NCT ID NCT03159026

First seen Jan 29, 2026 · Last updated May 16, 2026 · Updated 12 times

Summary

This study looked back at the medical records of 38 people with a specific genetic form of propionic acidemia, a rare metabolic disorder. The goal was to understand how the disease progresses over time, especially heart problems, and to create better guidelines for preventive care. No new treatments were tested; instead, researchers gathered information to help doctors manage the condition more effectively.

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Contacts and locations

Locations

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

Conditions

Explore the condition pages connected to this study.