Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects

Autor:	Massimo Russo, Luca Gentile, Antonio Toscano, M’Hammed Aguennouz, Giuseppe Vita, Anna Mazzeo
Jazyk:	angličtina
Rok vydání:	2020
Předmět:	ATTRv amyloidosis hereditary polyneuropathy TTR stabilizer TTR silencers gene therapy Neurosciences. Biological psychiatry. Neuropsychiatry RC321-571
Zdroj:	Brain Sciences, Vol 10, Iss 12, p 952 (2020)
Druh dokumentu:	article
ISSN:	2076-3425 72002441
DOI:	10.3390/brainsci10120952
Popis:	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 by regulatory agencies, since then the attention toward this disease has progressively increased and several drugs with different mechanisms of action are now available. This review describes the drugs already on the market, those that have shown interesting results although not yet approved, and those currently being tested.
Databáze:	Directory of Open Access Journals
Externí odkaz:	https://doaj.org/article/eb1ecf720024411b8765ac80c6a8b566 Zobrazit plný text záznamu View record in DOAJ Plný text ve formátu PDF Plný text ve formátu HTML
Nepřihlášeným uživatelům se plný text nezobrazuje	K zobrazení výsledku je třeba se přihlásit.