News

A single dose of gene-editing therapy nexiguran ziclumeran (nex-z) leads to deep reductions in blood levels of the disease-causing transthyretin (TTR) protein in adults with…

Yoltech Therapeutics has raised about $45 million in funding to advance its clinical programs, including YOLT-201, an experimental gene-editing therapy for familial amyloid…

There is a high prevalence of the Val30Met (V30M) TTR gene mutation — the most common mutation associated with familial amyloid polyneuropathy (FAP) and…

Hereditary transthyretin amyloidosis (ATTRv), a group of diseases that includes familial amyloid polyneuropathy (FAP), might not be as easily categorized into distinct neurological and…

Specialists in Scandinavia have updated the Kumamoto scale, a tool long used to monitor the progression of hereditary transthyretin amyloidosis (ATTRv), a group of diseases…

People with early-stage familial amyloid polyneuropathy (FAP) cost society more than twice as much as symptom-free carriers of disease-causing genetic mutations, largely due to…

Early genetic screening for people with unexplained nerve damage, or neuropathy, may help uncover undiagnosed cases of familial amyloid polyneuropathy (FAP), allowing faster access…

A Phase 3 clinical trial testing Intellia Therapeutics’ gene-editing therapy nexiguran ziclumeran (nex-z) in adults with familial amyloid neuropathy (FAP) is ahead of…

Using a small sample of tissue from skin — obtained via a skin biopsy — can help diagnose familial amyloid polyneuropathy (FAP) early by…

Kidney disease is an underrecognized complication of hereditary transthyretin amyloidosis (ATTRv) — a group of diseases that includes familial amyloid polyneuropathy (FAP) — but…