Problems with memory and problem-solving in people with hereditary transthyretin amyloidosis (ATTRv), a group of diseases that includes familial amyloid polyneuropathy (FAP), associated with a thinning in specific brain regions, according to a small brain imaging study. “The reasons for cognitive involvement in ATTRv are still a subject of…
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According to the Murdoch Children’s Research Institute, there are more than 7,000 rare genetic diseases in the world, mostly affecting children. Additionally, about 300 million people worldwide live with a rare disease, and most of these diseases have no approved treatment. These numbers underscore the urgent need for more research…
A single dose of nucresiran, an experimental therapy for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis, safely resulted in significant and sustained reductions in blood levels of the disease-driving transthyretin (TTR) protein in healthy people. That’s according to interim results from an ongoing, placebo-controlled Phase 1…
A mutation of the TTR gene that’s been exclusively reported in Chinese people causes an eye condition called vitreous opacity that can be successfully managed with eye surgery, but eventually recurs in nearly all cases after a mean of five years, a study shows. After surgery, which is called…
As a columnist for FAP News Today since October 2020, I’ve written about various topics, all related to my experience caring for a spouse who had hereditary ATTR amyloidosis. My columns touch on the emotional and physical challenges I faced as a caregiver, the adjustments we…
One year after a single dose of Intellia Therapeutics’ experimental gene-editing therapy NTLA-2001, blood levels of the disease-driving transthyretin (TTR) protein dropped by a mean of 91% in people with familial amyloid polyneuropathy (FAP). Available data at two years showed sustained TTR level reductions, which were accompanied…
Becoming educated and informed about FAP is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your FAP journey.
There is no cure for FAP yet, but there are treatments that can help manage the disease. Learn more about the approved and experimental therapies here.
