Familial amyloid neuropathy
MONDO:0007100A rare genetic systemic disease characterized by adult onset, progressive sensorimotor and autonomic neuropathy and infiltrative cardiomyopathy. Neurological involvement usually starts with sensory loss in the extremities and progresses with motor neuropathy. Cardiomyopathy presents with rhythm abnormalities and heart failure. The disease also frequently manifests with a range of additional clinical signs and symptoms due to associated ocular, renal, central nervous system and gastrointestinal involvement.
Also known as: ATTRv amyloidosis, amyloid neuropathies, familial, familial TTR-related amyloidosis, familial amyloid neuropathy, familial amyloid polyneuropathy, familial transthyretin-related amyloidosis, hATTR, hereditary TTR amyloid polyneuropathy
60 clinical trials for this condition and its sub-types.
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Broader categories
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Could a simple blood test track nerve damage? a study investigates NT-3 levels in neuropathy.
Knowledge-focused TerminatedThis study measures levels of a protein called NT-3 in the blood of people with peripheral neuropathy or Charcot-Marie-Tooth disease. Researchers will compare these levels with measures of muscle strength, mobility, and daily function. The goal is to see whether NT-3 levels corre…
Sponsor: Zarife Sahenk • Aim: Knowledge-focused
Last updated Jul 31, 2026 00:00 UTC
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Tafamidis tablet vs capsule: which works better?
Knowledge-focused TerminatedThis early-stage study aimed to compare how a tablet form of tafamidis is absorbed in the body compared to the existing capsule form. It involved 24 healthy adults who took a single dose of each form under fed conditions. The study was terminated early, so results may be limited.
Phase: PHASE1 • Sponsor: Pfizer • Aim: Knowledge-focused
Last updated Jun 28, 2026 00:00 UTC
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Hidden heart condition: study seeks to uncover missed diagnosis in heart failure patients
Knowledge-focused TerminatedThis study aims to find out how common transthyretin amyloidosis cardiomyopathy (ATTR-CM) is in Russian patients with a certain type of heart failure. Researchers will review medical records and then invite some patients for extra heart tests to confirm or rule out ATTR-CM. The g…
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:47 UTC