AMYLOIDOSIS, HEREDITARY
Clinical trials for AMYLOIDOSIS, HEREDITARY explained in plain language.
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One-Shot gene therapy aims to halt rare, devastating nerve disease
Disease control OngoingThis Phase 3 trial is testing whether a single dose of an experimental gene therapy called NTLA-2001 can safely slow or stop the progression of hereditary amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. About 50 participants worldwide will …
Matched conditions: AMYLOIDOSIS, HEREDITARY
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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Doctors probe heart impact of amyloidosis drugs
Knowledge-focused OngoingThis study aims to better understand how two existing drugs, patisiran and vutrisiran, affect the heart in patients with a rare inherited disease called hereditary transthyretin amyloidosis (ATTRv). Researchers will observe 20 patients who have both nerve and heart symptoms and a…
Matched conditions: AMYLOIDOSIS, HEREDITARY
Sponsor: Rennes University Hospital • Aim: Knowledge-focused
Last updated Apr 02, 2026 07:26 UTC