Steve Bryson, PhD, science writer —

Older age and worse disease were significantly associated with cognitive impairments in people with familial amyloid polyneuropathy (FAP) who underwent a liver transplant, a large study suggested. Researchers noted these cognitive findings are consistent with the natural history of the disease. “No distinct pattern was found for…

Eplontersen reduced transthyretin (TTR) protein levels, eased disease progression, and improved quality of life in adults with familial amyloid polyneuropathy (FAP), according to an eight-month interim analysis of the NEURO-TTRansform study. Based on these data, the investigational therapy’s co-developers Ionis Pharmaceuticals and AstraZeneca will seek regulatory approval…

Members of a Chinese family carrying a rare gene mutation causing familial amyloid polyneuropathy (FAP) experienced abnormal protein deposits in the eye — manifesting as “floaters” — before having any other noticeable disease symptoms, a case series reported. Such protein deposits can lead to functional abnormalities of the retina,…

A seven-year genetic screening program in Bulgaria involving people at high risk of hereditary transthyretin amyloidosis — a group of conditions that also includes familial amyloid polyneuropathy (FAP) — found patients showed mixed symptoms, with some clinical peculiarities related to specific disease-causing mutations. According to researchers, such genetic screening is…

The first patient has been dosed in the second part of a Phase 1 trial evaluating NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis. The first part of the study exposed patients to single ascending doses of…

Vutrisiran, an experimental therapy for familial amyloid polyneuropathy (FAP), continued to safely alleviate neurologic impairments and overall disability over 18 months of treatment, according to new data from the ongoing Phase 3 HELIOS-A trial. Those being given the therapy, administered as an under-the-skin injection once every three months,…

Ionis Pharmaceuticals has entered into a collaboration agreement with AstraZeneca to develop and commercialize eplontersen, Ionis’ investigational therapy to treat transthyretin amyloidosis (ATTR). ATTR amyloidosis is a group of conditions characterized by the formation of toxic aggregates or clumps of the TTR protein that build up in…

Vutrisiran, an investigational therapy for familial amyloid polyneuropathy (FAP), eased neurological impairments, lessened heart stress, and ameliorated quality of life, nutritional status, and social engagement in adult patients, according to new nine-month data from HELIOS-A trial. “These additional data from the HELIOS-A study show the potential of vutrisiran…

Novo Nordisk has acquired Prothena’s clinical-stage antibody PRX004, an investigational therapy for people with hereditary transthyretin amyloidosis (ATTR), which also includes familial amyloid polyneuropathy (FAP), as well as other non-hereditary forms of the disease. While the company initially will focus on developing PRX004 for the treatment of ATTR…

Living with hereditary transthyretin amyloidosis (hATTR), a group of rare disorders that includes familial amyloid polyneuropathy (FAP), affects the quality of life for both patients and their families, a large questionnaire study in Italy found. Among the more common and practical consequences for…