Ontology highlight
ABSTRACT:
SUBMITTER: Russo M
PROVIDER: S-EPMC7763612 | biostudies-literature | 2020 Dec
REPOSITORIES: biostudies-literature
Brain sciences 20201209 12
Hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy is a progressive disease that is transmitted as an autosomal dominant trait and characterized by multiple organ failure, including axonal sensory-motor neuropathy, cardiac involvement, and autonomic dysfunction. Liver transplantation (LT) and combined heart-liver transplantation, introduced in the 1990s, have been the only therapies for almost two decades. In 2011, tafamidis meglumine became the first specific drug approved ...[more]