WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS
Clinical trials for WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS explained in plain language.
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Gene-Editing breakthrough: single dose may stop rare fatal disease
Disease control CompletedThis early-stage trial tested a new gene-editing drug, NTLA-2001, in 72 adults with hereditary ATTR amyloidosis, a condition that causes nerve damage and heart problems. The goal was to see if a single dose is safe and can lower the abnormal protein (TTR) that builds up in the bo…
Matched conditions: WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS
Phase: PHASE1 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 26, 2026 10:14 UTC
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Promising early trial: patisiran may ease nerve damage in rare amyloidosis
Disease control CompletedThis early-phase study looked at how well the drug patisiran works for people with a rare disease called wild-type ATTR amyloidosis that causes nerve damage. Ten adults received the treatment, and researchers tracked changes in nerve function, symptoms like dizziness and digestio…
Matched conditions: WILD-TYPE TRANSTHYRETIN CARDIAC AMYLOIDOSIS
Phase: EARLY_PHASE1 • Sponsor: Austin Neuromuscular Center • Aim: Disease control
Last updated May 26, 2026 10:11 UTC