AMYLOID NEUROPATHIES, FAMILIAL

Clinical trials for AMYLOID NEUROPATHIES, FAMILIAL explained in plain language.

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Not yet finished but already full! 1 Completed 1

One-Shot gene therapy aims to halt rare, devastating nerve disease

Disease control Ongoing

This 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: AMYLOID NEUROPATHIES, FAMILIAL

Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control

Last updated Mar 31, 2026 12:12 UTC

AMYLOID NEUROPATHIES, FAMILIAL

Get alerted when new AMYLOID NEUROPATHIES, FAMILIAL trials appear

One-Shot gene therapy aims to halt rare, devastating nerve disease