AMYLOIDOSIS, FAMILIAL
Clinical trials for AMYLOIDOSIS, FAMILIAL explained in plain language.
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One-shot gene therapy aims to stop rare nerve disease in its tracks
Disease control Recruiting nowThis study tests a single dose of a gene therapy called NTLA-2001 in 60 people with a rare inherited nerve disease (ATTRv-PN). The goal is to see if it can safely reduce harmful protein buildup and slow nerve damage compared to a placebo. Participants must have a confirmed diagno…
Matched conditions: AMYLOIDOSIS, FAMILIAL
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 22, 2026 14:01 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