Hereditary amyloidosis
MONDO:0018634Hereditary amyloidosis refers to a group of inherited conditions that make up one of the subtypes of amyloidosis. Hereditary amyloidosisis characterized by the deposit of an abnormal protein called amyloid in multiple organs of the body where it should not be, which causes disruption of organ tissue structure and function. In hereditary amyloidosis, amyloid deposits most often occur in tissues of the heart, kidneys, and nervous system. While symptoms of hereditary amyloidosis may appear in childhood, most individuals do not experience symptoms until adulthood. There are many types of hereditary amyloidosis associated with different gene mutations and abnormal proteins. The most common type of hereditary amyloidosis is transthyretin amyloidosis (ATTR),a condition in which the amyloid deposits are most often made up of the transthyretin protein which is made in the liver. Other examplesof hereditary amyloidosis include, but are not limited to, apolipoprotein AI amyloidosis (A ApoAI), gelsolin amyloidosis (A Gel), lysozyme amyloidosis (A Lys), cystatin C amyloidosis (A Cys), fibrinogen Aα-chain amyloidosis (A Fib), and apolipoprotein AII amyloidosis (A ApoAII). Most types of hereditary amyloidosis are inherited in an autosomal dominant manner. Treatment is focused on addressing symptoms of organ damage and slowing down the production of amyloid when possible through methods such as liver transplants.
Also known as: amyloidosis, Familial, hereditary amyloidosis (disease), amyloidosis hereditary, familial amyloidosis
82 clinical trials for this condition and its sub-types, 19 tagged with Hereditary amyloidosis itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Hereditary amyloidosis
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Familial amyloid neuropathy 52 trials · 54 incl. sub-types
4 sub-types
- Amyloidosis, hereditary systemic 1 0 trials · 10 incl. sub-types Sub-types →
- Amyloidosis, hereditary systemic 3 0 trials
- Amyloidosis, hereditary systemic 5 0 trials
- Amyloidosis, hereditary systemic 6 0 trials
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Cerebral amyloid angiopathy 16 trials · 17 incl. sub-types
4 sub-types
- ACys amyloidosis 1 trial
- Cerebral amyloid angiopathy, APP-related 0 trials · 1 incl. sub-types Sub-types →
- ABri amyloidosis 0 trials
- ADan amyloidosis 0 trials
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APP-related brain and vascular amyloidosis 0 trials · 5 incl. sub-types
2 sub-types
- Alzheimer disease type 1 4 trials
- Cerebral amyloid angiopathy, APP-related 0 trials · 1 incl. sub-types Sub-types →
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3 sub-types
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Finnish type amyloidosis 0 trials
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ITM2B amyloidosis 0 trials
2 sub-types
- ABri amyloidosis 0 trials
- ADan amyloidosis 0 trials
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Familial visceral amyloidosis 0 trials
4 sub-types
- AApoAI amyloidosis 0 trials
- AFib amyloidosis 0 trials
- ALys amyloidosis 0 trials
- Apolipoprotein A-II amyloidosis 0 trials
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Pulmonary amyloidosis 0 trials
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Variant ABeta2M amyloidosis 0 trials
Most studied deeper sub-types
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New injection for rare protein disease passes first safety check
Disease control CompletedThis early-stage study tested a new drug called ALN-TTRSC04 in 96 healthy people to see if it is safe and how the body processes it. The drug aims to lower a harmful protein that causes transthyretin amyloidosis, a serious disease affecting nerves and the heart. Participants rece…
Phase 1 • Sponsor: Alnylam Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 09:06 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 …
Phase 3 • Sponsor: Alnylam Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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New PET tracer could spot rare heart disease without a biopsy
Diagnosis CompletedThis completed Phase 3 study tested whether a radioactive tracer called [18F]Florbetaben, used in PET scans, can accurately diagnose cardiac AL amyloidosis—a condition where abnormal proteins build up in the heart. 244 adults with suspected cardiac amyloidosis received a single P…
Phase 3 • Sponsor: Lantheus Germany GmbH • Aim: Diagnosis
Last updated Aug 05, 2026 00:00 UTC