WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS
Clinical trials for WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS explained in plain language.
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Can a new drug ease nerve pain in rare heart disease?
Disease control CompletedThis study tested the drug patisiran in 10 adults with a rare condition called ATTR amyloidosis that causes nerve damage. Participants received an IV infusion every 21 days for 24 months. Researchers measured changes in nerve function, autonomic symptoms, and quality of life to s…
Matched conditions: WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS
Phase: EARLY_PHASE1 • Sponsor: Austin Neuromuscular Center • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC
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Gene editing breakthrough? CRISPR therapy targets rare amyloidosis
Disease control CompletedThis early-stage trial tests NTLA-2001, a CRISPR-based gene editing therapy, in 72 adults with hereditary ATTR amyloidosis—a condition where abnormal protein builds up in nerves or the heart. The treatment aims to turn off the faulty gene to reduce protein levels. The study focus…
Matched conditions: WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS
Phase: PHASE1 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC