Select publications regarding antisense technology and TTR amyloidosis

Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy
Ackermann, E.J. et al. (2012). Amyloid. 19, 43-44.

Rate of Progression of Transthyretin Amyloidosis
Benson, M.D. et al. Am J Cardiol. 108, 285-289.

Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides
Benson, M.D. et al. Muscle Nerve. 33, 609-618.

ApoC-III (volanesorsen, FCS and FPL)

March 12, 2018
Inhibition of Apolipoprotein C-III with GalNAc-Conjugated Antisense Drug Potently Lowers Fasting Serum Apolipoprotein C-III and Triglyceride Levels in Healthy Volunteers with Elevated Triglycerides
Alexander et al. (Poster presentation) American College of Cardiology Annual Scientific Session & Expo.

September 11,  2017
The burden of familial chylomicronemia syndrome from the patients’ perspective
Gelrud et al. Expert Review of Cardiovascular Therapy

March 24, 2017
The burden of familial chylomicronemia syndrome: interim results from the IN-FOCUS study
Davidson et al. Expert Review of Cardiovascular Therapy

November 8, 2016
Building a better understanding of the burden of disease in familial chylomicronemia syndrome
Ahmad et al. Expert Review of Clinical Pharmacology

August 27, 2016
Antisense-Mediated Lowering of Plasma Apolipoprotein C-III by Volanesorsen Improves Dyslipidemia and Insulin Sensitivity in Type 2 Diabetes
Digenio et al. Diabetes Care

August 12, 2015
Shutting down the messenger: Antisense treatment for hypertriglyceridemia
Wang, Andrew Z. Science Translational Medicine

July 30, 2015
Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia
Gaudet et al. The New England Journal of Medicine

December 4, 2014
Targeting APOC3 in the Familial Chylomicronemia Syndrome
Gaudet et al. The New England Journal of Medicine

High Lipoprotein [LP(a)]

November 2, 2017
Temporal variability in lipoprotein(a) levels in patients enrolled in the placebo arms of IONIS-APO(a)Rx and IONIS-APO(a)-LRx antisense oligonucleotide clinical trials 
Marcovina et al. Journal of Clinical Lipidology

September 21, 2016
Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomised, double-blind, placebo-controlled, dose-ranging trials
Viney et al. The Lancet

September 15, 2015
Oxidized Phospholipids, Lipoprotein(a), and Progression of Calcific Aortic Valve Stenosis
Capoulade et al. The Journal of the American College of Cardiology

July 22, 2015
Antisense therapy targeting apolipoprotein(a): a randomized, double-blind, placebo-controlled phase 1 study
Tsimikas et al. The Lancet

Angiopoietin-like 3 [ANGPTL3]

July 20, 2017
Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides
Graham et al. The New England Journal of Medicine

June 14, 2017
Dyslipidaemia: ANGPTL3: a therapeutic target for atherosclerosis
Lim. Nature Reviews Cardiology