hATTR-PN

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…

Advocacy starts with patients and caregivers. It’s the husband who asks the nurse, “Could the lightheadedness mean something more?” It’s the daughter who brings journal articles…

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…

In my work as an advocate for the New Zealand Amyloidosis Patients Association (NZAPA), I meet many people with ATTR amyloidosis and their…

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…