News

‘Light the Night’ draws attention to Amyloidosis Awareness Month

The Amyloidosis Foundation again is marking Amyloidosis Awareness Month, observed each March, with a “Light the Night for Amyloidosis” campaign. The initiative seeks to call attention to amyloidosis, a group of diseases that includes familial amyloid polyneuropathy (FAP), a progressive disorder caused by the accumulation of amyloid fibrils…

Tafamidis found to increase survival over liver transplant in hATTR

Adults with hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), lived longer after treatment with tafamidis versus undergoing a liver transplant, a study reported. However, patients treated with tafamidis experienced a faster decline in neurological and heart-related outcomes than did those who…

Sudoscan noninvasive skin test may help improve early FAP diagnosis

Sudoscan, a commercial device that evaluates sweat gland function, might be a useful noninvasive tool to assess damage to small nerve fibers in people with familial amyloid polyneuropathy (FAP), a small study showed. Those small nerve fibers usually are affected in the early stages of the disease, leading to symptoms like…

FAP, hATTRs impact caregiver health-related quality of life: Survey

Caring for a person with hereditary transthyretin amyloidosis (hATTR), a group of conditions that includes familial amyloid polyneuropathy (FAP), has a considerable impact on caregivers’ health-related quality of life (HRQoL) and productivity, a survey reported. The findings were in the study, “A survey exploring caregiver burden…

Agency Recommends England’s NHS Cover Amvuttra for Treating FAP

England’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending Amvuttra (vutrisiran) be available through the country’s National Health Service (NHS) for treating familial amyloid polyneuropathy (FAP). Amvuttra is one of the first rare disease treatments to get the green light from NICE…

Small Nerve Fiber Problems Can Precede Symptoms in Late-onset FAP

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 amyloid polyneuropathy (FAP) before symptoms start, a small study suggested. Adults with disease-causing mutations for late-onset FAP but no disease symptoms had more abnormal responses…