siRNA Phase 2 Open-Label Extension Study for Treating Familial Amyloid Polyneuropathy
H Schmidt, T Coelho, O Suhr, I Conceicao, M Waddington-Cruz, J Buades, J Campistol, J Pouget, J Berk, J Gollob, D Adams · Zeitschrift für Gastroenterologie · 2015 · Heft 08
Bibliografische Angaben
Zusammenfassung
Einleitung: Familial amyloid polyneuropathy (FAP) is a progressive and fatal, autosomal dominant disease caused by deposition of mutant and wild-type transthyretin (TTR). Liver transplantation serves currently as therapeutic standard. Patisiran is an investigational systemically administered lipid nanoparticle formulation of a small interfering RNA (siRNA) targeting wild-type and mutant TTR. Material und Methoden: A Phase 2 open-label extension (OLE) study of patisiran in patients with FAP who participated in the Phase 2 patisiran trial, was initiated in October 2013. The primary objective of the study is to eva…
