TRANSTHYRETIN AMYLOIDOSIS
Clinical trials for TRANSTHYRETIN 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: TRANSTHYRETIN AMYLOIDOSIS
Phase: EARLY_PHASE1 • Sponsor: Austin Neuromuscular Center • Aim: Disease control
Last updated Jun 27, 2026 12:37 UTC
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New shot every 3 months could slow rare nerve disease
Disease control CompletedThis study tested a new drug called vutrisiran in 164 people with a rare inherited disease that damages nerves and organs. Participants received either vutrisiran as a shot every 3 months or the standard treatment patisiran as an IV infusion every 3 weeks. The goal was to see if …
Matched conditions: TRANSTHYRETIN AMYLOIDOSIS
Phase: PHASE3 • Sponsor: Alnylam Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Can MRI and nerve tests catch a rare disease before symptoms start?
Knowledge-focused CompletedThis study looked at whether advanced MRI scans and a nerve test called MUNIX can detect the earliest signs of nerve damage in people who carry a gene mutation for familial amyloid neuropathy. Researchers studied 60 adults with the TTR mutation, comparing those with and without s…
Matched conditions: TRANSTHYRETIN AMYLOIDOSIS
Phase: NA • Sponsor: Assistance Publique Hopitaux De Marseille • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:30 UTC
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Protein clues could spot rare disease years early
Knowledge-focused CompletedThis study followed 37 people with a genetic risk for hereditary ATTR amyloidosis over five years. Researchers measured levels of misfolded proteins in the blood to see if they could detect the earliest signs of the disease. The goal is to develop a way to catch the condition bef…
Matched conditions: TRANSTHYRETIN AMYLOIDOSIS
Sponsor: The Cleveland Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC