Lindsey Shapiro, PhD, science writer —

An international panel of patient advocates and healthcare providers has published the first set of recommendations outlining steps for the holistic care of people living with hereditary transthyretin-mediated amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP). The guidelines focus not only on early diagnosis…

AstraZeneca has added Latin America to the list of regions in which it has exclusive commercialization rights for eplontersen, an investigational therapy being reviewed for approval by the U.S. Food and Drug Administration (FDA) to treat familial amyloid polyneuropathy (FAP). Eplontersen’s original developer, Ionis…

Advanced MRI imaging of the sciatic nerve — a large bundle of nerve fibers running from the lower back down the length of the leg — may help to distinguish familial amyloid polyneuropathy (FAP) patients from healthy people, a study found. Such scans also may work to identify presymptomatic…

A U.S. Food and Drug Administration (FDA) advisory committee will meet Sept. 13 to discuss Alnylam Pharmaceuticals‘ application of Onpattro (patisiran) for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM), according to the company. Data from the placebo-controlled Phase 3 APOLLO-B clinical trial (NCT03997383) will be reviewed at…

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 disease spontaneously resolved for three men in the U.K. Accompanying ATTR-CM’s resolution was the presence in the bloodstream of antibodies targeted against the insoluble transthyretin…

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 in other nations, according to a recent study. From 2008-2020, FAP prevalence, or the total number of people living with the disease, in Taiwan ranged…

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 disease was stabilized by liver transplants, a study showed. In the reemergence of disease symptoms, the brothers showed also brain and eye problems that were…

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 amyloidosis with cardiomyopathy (ATTR-CM). A pivotal trial is one where the data are expected to support an application for a therapy’s regulatory approval. Intellia is…

Amvuttra (vutrisiran) and Onpattro (patisiran) showed similar safety and efficacy for familial amyloid polyneuropathy (FAP) patients, according to new analyses from the Phase 3 HELIOS-A trial that supported Amvuttra’s regulatory approval. Both therapies led to similar reductions in transthyretin, the protein that accumulates in FAP, and they were…

More than one year of treatment with eplontersen safely reduced symptoms of nerve damage and improved quality of life for adults with familial amyloid polyneuropathy (FAP), according to top-line data from the Phase 3 NEURO-TTRansform trial. The experimental therapy from Ionis Pharmaceuticals and AstraZeneca also significantly…