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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Can a daily pill keep a rare heart disease in check for the long haul?
Disease control Not yet recruitingThis study tests the long-term safety and tolerability of acoramidis, an oral medication that stabilizes the transthyretin protein, in people newly diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition that can lead to heart failure. Participants who complete…
Phase: PHASE3 • Sponsor: Eidos Therapeutics, a BridgeBio company • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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New scan spots hidden heart disease in gene carriers before symptoms start
Diagnosis Not yet recruitingThis study aims to see if a new radioactive dye can detect early signs of a heart condition called amyloidosis in people who carry a TTR gene mutation but have no heart failure symptoms. About 80 adults aged 30-80 will receive an injection of the dye and then have a PET/CT scan t…
Sponsor: University of Texas Southwestern Medical Center • Aim: Diagnosis
Last updated Jun 27, 2026 12:35 UTC
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AI spots heart clues in bone scans: a new way to catch a silent killer?
Diagnosis Not yet recruitingThis study tests whether a computer model can find signs of a heart condition called cardiac amyloidosis in bone scans that were done for other reasons. Researchers will look back at 57 patients' scans to see if the model can correctly identify those with the disease. If it works…
Sponsor: University Hospital, Lille • Aim: Diagnosis
Last updated Jun 27, 2026 12:33 UTC
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New PET scan could spot hidden heart disease
Diagnosis Not yet recruitingThis study tests whether a special PET-CT scan using a drug called evuzamitide can detect signs of cardiac amyloidosis in people whose standard heart scans were unclear but who have high levels of a blood marker called TAD1. About 25 adults with heart failure or a genetic risk fo…
Sponsor: Lorena Saelices • Aim: Diagnosis
Last updated Jun 27, 2026 12:09 UTC
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1,000 gene carriers tracked to unlock amyloidosis secrets
Knowledge-focused Not yet recruitingThis study is a registry that will collect health information from 1,000 people who carry the gene for hereditary amyloidosis, including those with and without symptoms. Researchers will track who develops the disease and how it progresses, including the need for heart transplant…
Sponsor: Virginia Commonwealth University • Aim: Knowledge-focused
Last updated Aug 14, 2026 00:00 UTC
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New study tracks safety of eplontersen in patients with liver transplants
Knowledge-focused Not yet recruitingThis observational study monitors the long-term safety of eplontersen, a treatment for transthyretin amyloidosis, in patients who have had a liver transplant or have severe liver impairment. Researchers will track side effects, lab changes, and serious health events in about 320 …
Sponsor: AstraZeneca • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC