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 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…
Discussion
Long-term treatment with Amvuttra (vutrisiran) was found to be safe and to sustain disease stability in people with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). That’s according to up to five years of data from…
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Long-term treatment with diflunisal may help slow disease progression in people with hereditary transthyretin amyloidosis (hATTR), according to a Swedish real-world study. The therapy is approved in the European Union (EU), but not…
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A computerized test that measures how quickly and accurately people perform finger tapping and hand movement tasks was able to detect subtle motor problems in people with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) earlier…
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People carrying the p.V142I mutation in the TTR gene — which is mostly associated with hereditary transthyretin amyloid cardiomyopathy (hATTR-CM) — often have polyneuropathy, or damage to multiple nerves, a U.S. study shows. But…
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Researchers have identified a common structure among toxic transthyretin (TTR) clumps, called amyloid fibrils, in heart tissue from people carrying mutations linked to hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). Such structural similarity contrasts with…
Discussion
Increased levels of neurofilament light chain (NfL), a marker of nerve damage, are associated with more severe neurological symptoms in people with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), according to a new study from…