Marisa Wexler, MS, senior science writer —

Treatment with Tegsedi (inotersen) eased a range of neuropathy symptoms in people with familial amyloid polyneuropathy (FAP), new data from the NEURO-TTR Phase 2/3 trial show. The findings, “Neuropathy Symptom and Change: Inotersen Treatment of Hereditary Transthyretin Amyloidosis,” were published in the journal Muscle & Nerve,…

Treatment with Tegsedi (inotersen) slows neurological disease progression in people with familial amyloid polyneuropathy (FAP), results from a Phase 2/3 clinical trial show. The findings may be useful in designing optimal tools for measuring response to treatment in FAP, researchers said. The study, “Mnis+7 And…

Levels of calprotectin, a marker of inflammation, are elevated in the feces of people with familial amyloid polyneuropathy (FAP) who experience gastrointestinal (GI) symptoms, a study suggests. This finding implicates inflammation as a possible cause of gut symptoms in FAP and supports measuring calprotectin levels in people with…

The Italian Medicines Agency has added Tegsedi (inotersen), a treatment for familial amyloid polyneuropathy (FAP), to the list of reimbursed therapies in the country. Tegsedi was approved to treat stage 1 or 2 polyneuropathy in adults with FAP — also called hereditary transthyretin (hATTR) amyloidosis…

Treatment with Onpattro (patisiran) improves multiple aspects of quality of life in people with familial amyloid polyneuropathy (FAP), findings from a Phase 3 clinical trial show. The rapid deterioration seen in participants given a placebo illustrate the importance of starting FAP treatment as early as possible, scientists say.

A type of surgery that involves removal of the eye’s “gel” can improve vision in people with familial amyloid polyneuropathy (FAP), a new study suggests. However, subsequent surgeries often are necessary to preserve vision, highlighting the need for close follow-up after surgery. The study, “Small gauge…

Long-term treatment with Vyndaqel (tafamidis) may reduce the risk of death in people with familial amyloid polyneuropathy (FAP), a study shows. The study, “Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years,” were…

Onpattro (patisiran) has been approved in Brazil to treat hereditary transthyretin amyloidosis in adults with stage 1 or stage 2 polyneuropathy, the therapy’s developer, Alnylam Pharmaceuticals, has announced. Hereditary ATTR amyloidosis, also known as familial amyloid polyneuropathy (FAP), is a genetic…

The European Commission (EC) has approved the once-daily oral medication Vyndaqel (tafamidis) for the treatment of cardiomyopathy — a disease of the heart muscle that makes it harder for the heart to pump blood to the rest of the body — in adults with transthyretin…

The protein cathepsin E — known for regulating immune system cells — may be a potential biomarker for familial amyloid polyneuropathy (FAP), a study suggests. Specifically, researchers found that the protein was present at comparatively low levels in the macrophages — a type of immune cells —…