Patients with hereditary ATTR amyloidosis, also called familial amyloid polyneuropathy (FAP), might soon have a new medication to look forward to, as data from a recent Phase 3 trial of patisiran showed the treatment improved quality of life and reduced the impairment caused by nerve damage. Alnylam Pharmaceuticals and…
A new study outlines the design and rationale for APOLLO, a Phase 3 clinical trial (NCT02510261) to evaluate the effect of patisiran (ALN-TTR02) in patients with hATTR (hereditary ATTR) amyloidosis with polyneuropathy. The study, “Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in…
Body-wide imaging can detect amyloid protein deposits in the organs of people with familial amyloid polyneuropathy (FAP), making it possible to assess the effects of new therapies without performing invasive tissue biopsies, a Japanese study reports. Imaging can also be used to see whether drugs that remove amyloid build-ups…
Researchers presented the main features of primary amyloidosis, showing the need for careful diagnosis in patients that present with neuropathic symptoms, especially autonomic neuropathy, to correctly identify other conditions such as familial amyloid neuropathy (FAP). The study, titled “Autonomic Neuropathy and Albuminocytologic Dissociation in Cerebrospinal Fluid As the Presenting…
An interim analysis of the Phase 3 trial for tafamidis (Fx-1006A) as a therapy for patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP) shows the investigational treatment delays disease progression, Pfizer announced. The study, “Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid…
People at risk for familial amyloid polyneuropathy (FAP) caused by mutations in the TTR gene have different levels of knowledge about their disease than people at risk of treatable genetic conditions, according to researchers at the European University of Lisbon. This is, however, not exclusive to people with a…
Chinese researchers report the case of a family affected by familial amyloid polyneuropathy (FAP) caused by a less common mutation in the transthyretin (TTR) gene. Their findings might offer scientists new understanding of the disease processes in FAP, and have implications for genetic counseling, researchers said. The study,…
Patisiran (ALN-TTR02) led to an improvement in average nerve dysfunction in 26 patients with familial amyloid polyneuropathy (FAP), a rare disease that causes organ or nerve malfunction. Moreover, all 25 eligible participants in the Phase 2 open label extension (OLE) study of patisiran were enrolled in the Phase 3 APOLLO…
GlaxoSmithKline (GSK) has backed away from the chance to license two Ionis Pharmaceuticals treatments for familial amyloid polyneuropathy (FAP) — also known as TTR amyloidosis (ATTR) — just as Ionis prepares to file for U.S. and European approval of inotersen, one of the two drugs. The move, which also…
A new clinical trial called TRAM aims to determine the prevalence of genetic mutations in the transthyretin (Ttr) gene, the underlying cause of TTR familial amyloid polyneuropathy (TTR-FAP). The trial (NCT03237494) is for adult patients with polyneuropathy disease or cardiomyopathy of unknown causes and is currently recruiting participants. Mutations…