A Phase 3 clinical trial that’s testing the oral treatment acoramidis to prevent or delay disease onset is continuing to recruit adults who carry a mutation known to cause hereditary transthyretin amyloidosis (hATTR), including familial amyloid polyneuropathy (FAP), but aren’t having symptoms yet. That’s according to a recent update…
Read more
I’ve just returned from an amyloidosis conference in Baveno, Italy, organized by the Amyloidosis Alliance. This biennial event — called the International ATTR Amyloidosis Meeting for Patients and Doctors — brings together patients, families, patient advocates, clinicians, and pharmaceutical companies from around the world for two days of…
Amvuttra (vutrisiran) appears to be safe for long-term use in adults with familial amyloid polyneuropathy (FAP) and those with another form of transthyretin amyloidosis (ATTR) that causes heart damage. That’s according to an analysis of more than 700 patients who received Amvuttra in one of two Phase 3…
A single dose of gene-editing therapy nexiguran ziclumeran (nex-z) leads to deep reductions in blood levels of the disease-causing transthyretin (TTR) protein in adults with familial amyloid polyneuropathy for up to three years. That’s according to longer-term data from a Phase 1 clinical trial (NCT04601051). The trial also…
Yoltech Therapeutics has raised about $45 million in funding to advance its clinical programs, including YOLT-201, an experimental gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of transthyretin amyloidosis (ATTR). YOLT-201 is being tested in a Phase 1/2a clinical trial (NCT06539208) that is still recruiting…
There is a high prevalence of the Val30Met (V30M) TTR gene mutation — the most common mutation associated with familial amyloid polyneuropathy (FAP) and other forms of hereditary transthyretin amyloidosis (ATTRv) — in Spain’s Balearic Islands, according to a new report by scientists in the region. The scientists believe…
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.
