News

A one-time gene-editing therapy from Intellia Therapeutics led to a sustained, near-complete reduction in blood levels of the disease-causing transthyretin (TTR) protein in adults…

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…

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…

The U.S. Food and Drug Administration (FDA) has extended the approval of Amvuttra (vutrisiran) to adults with cardiomyopathy (heart damage) due to nonhereditary or hereditary…