Top 10 FAP stories of 2024

Throughout 2024, FAP News Today informed our readers about the latest developments in treatment and clinical trials for familial amyloid polyneuropathy (FAP). Here is a list of the top 10 most read stories we published in 2024, along with a brief summary. We look forward to continuing to…

FDA approves eplontersen, now Wainua, for treating FAP in adults

The U.S. Food and Drug Administration (FDA) has approved eplontersen for treating familial amyloid polyneuropathy (FAP). The therapy, which will be sold under the brand name Wainua by its co-developers Ionis Pharmaceuticals and AstraZeneca, is specifically indicated for adults with the genetic disease. In a previous Phase…

Blood NfL levels may mark the onset of symptoms in FAP

Blood levels of neurofilament light chain (NfL) — a marker of nerve cell damage — can separate people with symptoms of forms of hereditary ATTR amyloidosis (hATTR), including familial amyloid polyneuropathy (FAP), from those who haven’t developed symptoms yet. That’s according to a study in Italy that also showed…

Detecting touch, temperature may help predict FAP onset

Testing the ability to sense temperatures and mechanical forces could help predict when symptoms will appear for people who carry mutations that cause familial amyloid polyneuropathy (FAP). That’s according to “Quantitative sensory testing and skin biopsy findings in late-onset ATTRv presymptomatic carriers: Relationships with predicted time of disease…

Noninvasive eye imaging technique may aid early FAP detection

Changes in the nerve fibers of the cornea — the eye’s clear protective outer layer — may help in the early diagnosis of hereditary transthyretin amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP), a study shows. A noninvasive imaging method, called corneal confocal microscopy (CCM), identified…

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…

UC San Diego Health 1st in US to Offer Amvuttra for FAP

The newly approved familial amyloid polyneuropathy (FAP) treatment Amvuttra (vutrisiran) is being offered at UC San Diego Health to help mitigate symptoms resulting from the nerve damage that marks the progressive disorder. According to UC San Diego Health, it’s the nation’s first health system to offer the treatment to…