hATTR-PN

Long-term oral treatment with tafamidis meglumine safely helped maintain nerve function, quality of life, nutritional status, and mobility in people living with familial…

A protein called glial fibrillary acidic protein (GFAP) is found at higher levels in the blood of people with familial amyloid polyneuropathy (FAP), even…

Sudoscan, a test used to indirectly assess small nerve fiber damage through sweat gland function, may be a marker of disease severity and progression in…

Family members of people with late-onset familial amyloid polyneuropathy (FAP) may face emotional and practical challenges due to the onset of disease symptoms in…

Here in New Zealand, clinicians and advocates are pushing our country’s government to update its genetic editing and modification legislation. Historically, New Zealand…

Tafamidis meglumine safely and effectively slows disease progression in most people living with familial amyloid polyneuropathy (FAP), according to a real-world study from…

The first adult with familial amyloid polyneuropathy (FAP) has been dosed in a Phase 3 clinical trial testing nexiguran ziclumeran (nex-z), Intellia Therapeutics’ one-time…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ART001, a gene editing candidate that Accuredit Therapeutics is developing as…

Levels of a blood protein called NfL may be useful as a marker of disease activity in people with familial amyloid polyneuropathy (FAP), a…

Wainzua (eplontersen) is now approved in the European Union (EU) for treating adults with early-stage familial amyloid polyneuropathy (FAP). With the European Commission’s approval, Wainzua…