News

ATTR amyloidosis with cardiomyopathy (ATTR-CM), noting damage to the heart, is usually considered to be an irreversible condition. But in a first known reporting, the…

Treatment with Onpattro (patisiran) for 1.5 years leads to sustained reductions in disability progression among people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or damage…

The prevalence of familial amyloid polyneuropathy (FAP) in Taiwan in recent years is consistent with other mid-to-high prevalence countries, but survival rates are lower than…

Onpattro (patisiran), a liver-directed gene-silencing therapy, successfully slowed progression of symptoms that emerged in two brothers with hereditary transthyretin amyloidosis (hATTR) decades after their…

Intellia Therapeutics is planning to launch pivotal clinical trials of its gene-editing therapy NTLA-2001 in people with familial amyloid polyneuropathy (FAP) and ATTR…

Amvuttra (vutrisiran) and Onpattro (patisiran) showed similar safety and efficacy for familial amyloid polyneuropathy (FAP) patients, according to new analyses from the Phase…

Treatment with Onpattro (patisiran) or Amvuttra (vutrisiran) leads to a reduction in blood levels of neurofilament light chain (NfL), a nerve cell damage…

People with hereditary transthyretin amyloidosis (hATTR) who are treated with a combination of therapies appear to have better health outcomes than those given only one…

Changes in the nerve fibers of the cornea — the eye’s clear protective outer layer — may help in the early diagnosis of hereditary transthyretin…

In the U.S., one-third of people with hereditary transthyretin amyloidosis (hATTR) — a group of disorders that includes familial amyloid polyneuropathy (FAP) — are diagnosed…