Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloid polyneuropathy (FAP), is a rare genetic disease characterized by damage to the peripheral nerves found outside the brain and spinal cord. It is typically…
Problems associated with nerve damage are the main symptoms of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). However, because the disease can affect multiple organs and systems in the body, including the heart, clinical manifestations…
Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) diagnosis typically involves identifying hATTR-PN symptoms and performing specialized testing to confirm the disease’s presence. Inherited mutations in the TTR gene cause hATTR-PN by destabilizing and changing the…
Medications, clinical monitoring, and symptom management can all play a role in hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) treatment. Also known as familial amyloid polyneuropathy, hATTR-PN is a genetic condition marked by polyneuropathy, or…
Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloid polyneuropathy (FAP), is a form of familial amyloidosis caused by mutations in the TTR gene, which contains instructions for cells to produce a…
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Discussion
A newly developed questionnaire may help flag early signs of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) in people carrying mutations in the TTR gene that are known to cause the disease, a study shows. Although…
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Early-career researchers studying transthyretin amyloidosis (ATTR), a group of conditions that includes hereditary ATTR with polyneuropathy (hATTR-PN) and ATTR cardiomyopathy (ATTR-CM), are being called to apply for one of two $100,000 research grants.
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A family history of nerve disease, carpal tunnel syndrome in both hands, and balance problems are the “clinical ‘red flags'” most strongly linked to a positive genetic test for mutations associated with hereditary transthyretin amyloidosis…
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Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) should be treated as early as possible once a patient has symptoms and a confirmed disease-causing genetic mutation, according to consensus recommendations from German experts. In addition, the…
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Although mutations that cause hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) most frequently lead to nerve damage, they can also cause heart damage and other symptoms, a study highlights. The findings “emphasize the need for…
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A specific genetic variant may have contributed to signs of severe liver problems observed in a few patients given nexiguran ziclumeran (nex-z), an investigational gene-editing therapy for hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) and the…
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A real-world study in Spain comparing two similar medications for hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) showed greater benefits with the infusion therapy Onpattro (patisiran) versus the injection treatment Tegsedi (inotersen) among…
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A type of cell damage called oxidative stress may be key to the development of disease symptoms in people who carry the most common TTR gene mutation that causes hereditary transthyretin amyloidosis with polyneuropathy…
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Measuring blood levels of a protein called neurofilament light chain (NfL) may help identify nerve damage in people with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) caused by the Val30Met mutation, the most common mutation…
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Amvuttra (vutrisiran) rapidly and consistently reduces blood levels of transthyretin (TTR), the disease-causing protein in hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), across a wide range of patients, according to clinical trial data. Similar…