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…
Latest News & Community Perspectives
The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on a Phase 3 clinical trial testing the investigational gene-editing therapy, nexiguran ziclumeran (nex-z), in adults with hereditary transthyretin amyloidosis with polyneuropathy…
More than half of adults with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), a condition marked by heart damage, also have multi-nerve damage, according to a new study from the University of Toronto. The findings…
MRI scans detect brain changes in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) before symptoms appear, according to a study. While these changes were not associated with cognitive problems in early-stage hATTR-PN patients…
Discussion
A diagnosis of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) between the ages of 30 and 60 can be profoundly life-altering. The gravity of the disease coincides with a period traditionally devoted to building, enjoying,…
Nerve ultrasound — a scan that uses high-frequency sound waves to create real-time images of the body’s nerves — may be used to monitor signs of polyneuropathy, or widespread damage, in people who carry disease-causing…
Adults who carry the Val30Met, or V30M, mutation — the most common cause of early-onset hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) — often develop heart rhythm problems early on, a study shows. Although V30M carriers…
Discussion
Hereditary diseases not only affect the body, but they also ripple through families, shaping relationships, identities, and mental health. I know this reality all too well. My late husband, Aubrey, was diagnosed in 2013…
RNA-targeted therapies — ones that zero in on molecules acting as messengers for genetic information in cells — are safe and effective at slowing the progression of neurological symptoms and improving quality of life among…
Polyneuropathy, or widespread nerve damage, occurs in both hereditary and wild-type transthyretin amyloidosis (ATTR), but the hereditary form, which includes familial amyloid polyneuropathy (FAP), tends to cause more severe nerve impairment. Hereditary ATTR was…
Undergoing a liver transplant and/or receiving disease-modifying treatments significantly improves survival in people with hereditary transthyretin amyloidosis (hATTR), a group of diseases that includes hATTR with polyneuropathy, or familial amyloid polyneuropathy (FAP). Disease-modifying drugs…