News

1 in 4 with ATTR amyloidosis have neurological and heart symptoms

New 15-year data from a global study involving more than 6,000 people with ATTR amyloidosis, an umbrella term for conditions that include familial amyloid polyneuropathy (FAP), found that nearly 1 in 4 patients experience both neurological and cardiac symptoms, showing the need, according to researchers, for improved multidisciplinary…

Unconventional tests detect FAP in 12 of 38 asymptomatic carriers

Diagnostic tests outside those considered standard for familial amyloid polyneuropathy (FAP) detected FAP in 12 of 38 people who carried disease-causing mutations but were asymptomatic, or without evident FAP symptoms, a study reported. The assessment methods, which identified FAP features when standard tests did not, included modified nerve…

Wainua now available in US through Orsini specialty pharmacy

A few weeks after its approval in the U.S., Wainua (eplontersen) will now be available to adults with familial amyloid polyneuropathy (FAP). Orsini was selected as the exclusive specialty pharmacy partner to provide Wainua, which was co-developed by Ionis Pharmaceuticals and AstraZeneca. The medication will also be made available through integrated…

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…

Eplontersen may stabilize, slow heart damage in FAP patients

A little more than a year of treatment with eplontersen was found to stabilize or even improve the heart’s structure and function in people with familial amyloid polyneuropathy (FAP) who also were experiencing symptoms of heart disease, known as cardiomyopathy. That’s according to an exploratory analysis of data…

Advanced screening may lead to early DMT start for ATTRv carriers

Screening for toxic protein clumps using biopsies and advanced imaging techniques may help people carrying mutations that cause hereditary transthyretin amyloidosis (ATTRv) — a group of conditions that includes familial amyloid polyneuropathy (FAP) — to get an early start on disease-modifying treatment (DMT). That’s according to a study in…

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…

Ultrasound may help assess FAP peripheral nerve malfunction

An ultrasound of the peripheral nerves could help monitor disease progression in people with early-stage familial amyloid polyneuropathy (FAP) who carry V30M, the most common disease-causing mutation. That’s according to a study in Sweden that also showed that while about half of patients analyzed showed no signs of peripheral…