News

  The number of people diagnosed with transthyretin amyloidosis (ATTR) in the U.S. is climbing, a surge researchers attribute to better medical awareness and more advanced diagnostic tools. This increase spans both new diagnoses and the total number of people living with the condition, which includes hereditary ATTR with…

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 TTR mutations but have not yet developed symptoms of hereditary transthyretin amyloidosis with polyneuropathy…

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 may be less likely to have cardiomyopathy, or heart muscle disease, those who do experience…

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 people with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). Those are the findings of a…

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 also more often associated with gastrointestinal symptoms and less often connected to carpal tunnel…

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 were significantly associated with a 78% lower chance of death, while liver transplant was linked…

Onpattro (patisiran) safely and effectively helps adults with familial amyloid polyneuropathy (FAP) caused by a V122I or T60A genetic mutation keep their disease from getting worse, according to a real-world study in the U.S. The post-marketing Phase 4 study (NCT04201418) focused on patients with one of these…

Alnylam Pharmaceuticals has launched a Phase 3 clinical trial testing its experimental treatment nucresiran in adults with familial amyloid polyneuropathy (FAP), also known as hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN). Top-line results are expected in 2028. This update came in the company’s latest pipeline and financial report, announced…

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 familial amyloid polyneuropathy (FAP) and the related condition ATTR amyloidosis with cardiomyopathy (ATTR-CM). The announcement came from Intellia Therapeutics, the therapy’s developer, less…

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 on hold after a participant developed potentially life-threatening liver damage. The participant received the therapy on Sept. 30 as part of the Phase 3…