AMYLOIDOSIS, HEREDITARY
Clinical trials for AMYLOIDOSIS, HEREDITARY 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, HEREDITARY
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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Tiny blood particles may reveal hidden heart disease
Knowledge-focused Recruiting nowThis study looks at whether tiny particles released by cells into the blood, called extracellular vesicles, can help detect heart damage from ATTR amyloidosis earlier and less invasively. Researchers will compare these particles in 70 people with different stages of the disease a…
Matched conditions: AMYLOIDOSIS, HEREDITARY
Sponsor: University of Sao Paulo General Hospital • Aim: Knowledge-focused
Last updated May 22, 2026 13:50 UTC