AMYLOIDOSIS, FAMILIAL
Clinical trials for AMYLOIDOSIS, FAMILIAL explained in plain language.
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Gene-Editing shot aims to halt rare nerve disease in phase 3 trial
Disease control Recruiting nowThis study tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a genetic disease that damages nerves. The goal is to see if the treatment can stop or slow the disease by lowering a harmfu…
Matched conditions: AMYLOIDOSIS, FAMILIAL
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 06, 2026 16:02 UTC
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New study seeks to unravel mysteries of rare, inherited disease
Knowledge-focused Recruiting nowThis study is observing 20 adults with a confirmed genetic form of ATTR amyloidosis, a serious condition where abnormal proteins build up in organs. The goal is to better understand the different ways the disease affects people and what factors influence its progression. Particip…
Matched conditions: AMYLOIDOSIS, FAMILIAL
Sponsor: Hospital de Alta Complejidad en Red • Aim: Knowledge-focused
Last updated May 07, 2026 18:41 UTC