TTR gene silencing therapy in post liver transplant hereditary ATTR amyloidosis patients.
Orly Moshe-LilieDiana DimitrovaStephen B HeitnerThomas H BrannaganSasha ZivkovicMazen HannaAhmad MasriMichael PolydefkisJohn L BerkMorie A GertzChafic KaramPublished in: Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis (2020)
TTR gene silencing therapy in hATTR patients with POLT could be a treatment option. Vigilant monitoring of renal, liver and bone marrow functions is necessary because of frequent complications. Further studies are needed to determine efficacy.