News

RNA-targeted therapies — ones that zero in on molecules acting as messengers for genetic information in cells — are safe and effective at slowing the…

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),…

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…

Onpattro (patisiran) safely and effectively helps adults with familial amyloid polyneuropathy (FAP) caused by a V122I or T60A genetic mutation keep their disease…

Alnylam Pharmaceuticals has launched a Phase 3 clinical trial testing its experimental treatment nucresiran in adults with familial amyloid polyneuropathy (FAP), also known…

An elderly man who developed life-threatening liver damage after receiving nexiguran ziclumeran (nex-z), an experimental gene-editing therapy, has died. The treatment was being tested for…

Two trials testing nexiguran ziclumeran (nex-z), a gene-editing therapy for familial amyloid polyneuropathy (FAP) and the related condition ATTR amyloidosis with cardiomyopathy (ATTR-CM), are…

Treatment switches in people with familial amyloid polyneuropathy (FAP) are common, with Amvuttra (vutrisiran) emerging as a preferred option due to its more…

A Phase 3 clinical trial that’s testing the oral treatment acoramidis to prevent or delay disease onset is continuing to recruit adults who carry a…

Amvuttra (vutrisiran) appears to be safe for long-term use in adults with familial amyloid polyneuropathy (FAP) and those with another form of transthyretin…