Protein clues may predict onset of rare genetic disease

NCT ID NCT03431896

First seen Feb 28, 2026 · Last updated May 14, 2026 · Updated 14 times

Summary

This study followed 37 people who carry a genetic mutation for hereditary ATTR amyloidosis but do not yet have symptoms. Over five years, researchers measured a specific misfolded protein in the blood to see if it changes when the disease starts or when treatment is needed. The goal was to find a way to detect early disease progression, not to test a treatment.

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Contacts and locations

Locations

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

Conditions

Explore the condition pages connected to this study.