News

The clinical symptoms of hereditary transthyretin amyloidosis (hATTR) — a group of disorders that includes familial amyloid polyneuropathy (FAP) — are highly influenced by the…

Onpattro (patisiran), an approved treatment for familial amyloid polyneuropathy (FAP), is now listed on Australia’s Pharmaceutical Benefits Scheme (PBS), meaning that eligible patients…

Young adults at risk for familial amyloid polyneuropathy (FAP) face unique psychological and social challenges, and likely would benefit from tailored healthcare support when…

A man carrying p.Val142Ile, a TTR gene mutation often linked to a condition related to familial amyloid polyneuropathy (FAP) but causing heart damage instead…

The first patient has been dosed in a China-based Phase 1/2a clinical trial testing YOLT-201, Yoltech Therapeutics’ one-time gene-editing therapy, in people with hereditary…

A second, therapeutic dose of Intellia Therapeutics‘ experimental gene-editing therapy NTLA-2001 safely and effectively reduced levels of the harmful transthyretin (TTR) protein in people…

The oral medication tafamidis meglumine appears to be well tolerated by adults with familial amyloid polyneuropathy (FAP) in France, where the treatment is…

Taiwanese people with familial amyloid polyneuropathy (FAP) have a significantly altered composition of intestinal bacteria relative to their unrelated healthy family members, a new…

Getting genetic counseling can encourage people at risk for hereditary transthyretin amyloidosis to have genetic testing and know they have the option to seek early…

Rising levels of a protein called neurofilament light chain (NfL) in the blood may indicate nerve damage due to familial amyloid polyneuropathy (FAP) even…