News

Tegsedi (inotersen) helped to stabilize familial amyloid polyneuropathy (FAP) symptoms in a 71-year-old man, but its use also was associated with a rare case of…

England’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending Amvuttra (vutrisiran) be available through the country’s National Health…

Eye involvement can become common over time in people with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP),…

An elderly man in Germany was found to carry one copy of a new mutation in the TTR gene that caused both neurological and heart…

Quantitative sensory testing (QST), a tool for monitoring nerve function, may help to detect problems in small nerve fibers in people with late-onset familial…

Alnylam Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to approve Onpattro (patisiran) for the…

A surgical procedure called suture trabeculotomy (SLOT) ab interno successfully treated glaucoma in nearly half of the eyes of familial amyloid polyneuropathy (FAP) patients treated…

Novo Nordisk is moving forward on a global Phase 2 clinical trial evaluating its experimental treatment NNC6019 in people with hereditary transthyretin amyloidosis (ATTR)…

Glu89Lys, a rare gene mutation that can cause familial amyloid polyneuropathy (FAP) — and is associated with early onset disease and a mix of…

One-time treatment with the experimental gene-editing therapy NTLA-2001 led to substantial reductions in the levels of toxic transthyretin protein that were sustained for several months…