News

The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ Onpattro (patisiran) as the first-ever treatment for patients in the U.S. with familial…

Evaluating the activity of sweat glands provides valuable information to assess peripheral nerve cell damage and help diagnose nerve diseases known as peripheral neuropathies, which…

The hereditary nature of familial amyloid polyneuropathy (FAP), along with the severity and unpredictability of the disease, highlights the risk for great psychological stress among…

Treatment with 20 mg a day of oral Vyndaqel (tafamidis) is enough for the therapy to reach the central nervous system (CNS) and eyes…

Next-generation sequencing to analyze a panel of 11 specific genes may facilitate the diagnosis of amyloidosis, including familial amyloid polyneuropathy (FAP), according to researchers…

Alnylam Pharmaceuticals’ investigative patisiran treatment improves overall health and prevents worsening of walking ability in patients with familial amyloid polyneuropathy (FAP), according to a…

Familial amyloid polyneuropathy (FAP) patients who do not have a Val30Met mutation exhibit prevalent cardiac and blood vessel dysfunction, but proper blood vessel constriction remains…

Treatment with the investigative therapy Tegsedi (inotersen) provides early and significant improvements in neuropathy symptoms and quality of life in patients with familial amyloid…

The European Commission (EC) has approved Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, to treat stage 1 or 2 polyneuropathy in adults with hereditary…

A Phase 3 trial testing patisiran as a therapy for hereditary ATTR amyloidosis with polyneuropathy (also called familial amyloid polyneuropathy, or FAP), showed…