METABOLISM, INBORN ERRORS

Clinical trials for METABOLISM, INBORN ERRORS explained in plain language.

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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: METABOLISM, INBORN ERRORS

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

Last updated Mar 31, 2026 12:12 UTC

METABOLISM, INBORN ERRORS

Get alerted when new METABOLISM, INBORN ERRORS trials appear

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