New hope for rare nerve disease? early trial tests patisiran

NCT ID NCT05023889

First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 16 times

Summary

This early-phase study tests the drug patisiran in 10 adults with a rare disease called wild-type ATTR amyloidosis that causes nerve damage. The goal is to see if patisiran can improve nerve function and quality of life over 24 months. Participants will have regular check-ups and questionnaires to track changes in symptoms like weakness, dizziness, and bowel issues.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Austin Neuromuscler Center/National Neuromuscular Research Institute

    Austin, Texas, 78759, United States

Conditions

Explore the condition pages connected to this study.