Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Rapid progression of familial amyloidotic polyneuropathy

(2015) David Adams et al.   NEUROLOGY

Liver Transplantation for Hereditary Transthyretin Amyloidosis

(2015) Bo-Göran Ericzon et al.   TRANSPLANTATION

Repurposing Diflunisal for Familial Amyloid Polyneuropathy

(2015) John L. Berk et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Effect on disability and safety of Tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy

(2013) P. Lozeron et al.   EUROPEAN JOURNAL OF NEUROLOGY

Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy

(2013) Teresa Coelho et al.   JOURNAL OF NEUROLOGY

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

(2013) Kevin Fitzgerald et al.   LANCET

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial

(2012) T. Coelho et al.   NEUROLOGY

Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy

(2012) T. Yamashita et al.   NEUROLOGY

Activation of complement by therapeutic liposomes and other lipid excipient-based therapeutic products: Prediction and prevention

(2011) Janos Szebeni et al.   ADVANCED DRUG DELIVERY REVIEWS

Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30)

(2010) Gösta Holmgren et al.   CLINICAL GENETICS

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Progression of transthyretin amyloid neuropathy after liver transplantation

(2010) J. J. Liepnieks et al.   NEUROLOGY

Cardiac amyloidosis in African Americans: Comparison of clinical and laboratory features of transthyretin V122I amyloidosis and immunoglobulin light chain amyloidosis

(2009) Lawreen H. Connors et al.   AMERICAN HEART JOURNAL

Liver transplantation for familial amyloidotic polyneuropathy: Impact on Swedish patients' survival

(2009) Sadahisa Okamoto et al.   LIVER TRANSPLANTATION

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates

(2008) M. Frank-Kamenetsky et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA