Search details
1.
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1.
N Engl J Med
; 384(13): 1216-1226, 2021 04 01.
Article
in English
| MEDLINE | ID: mdl-33789010
2.
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
N Engl J Med
; 382(24): 2289-2301, 2020 06 11.
Article
in English
| MEDLINE | ID: mdl-32521132
3.
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
N Engl J Med
; 380(6): 549-558, 2019 02 07.
Article
in English
| MEDLINE | ID: mdl-30726693
4.
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
Drug Metab Dispos
; 50(6): 781-797, 2022 06.
Article
in English
| MEDLINE | ID: mdl-34154993
5.
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.
N Engl J Med
; 379(1): 11-21, 2018 07 05.
Article
in English
| MEDLINE | ID: mdl-29972753
6.
Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.
Nucleic Acids Res
; 47(7): 3306-3320, 2019 04 23.
Article
in English
| MEDLINE | ID: mdl-30820542
7.
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis.
Circulation
; 139(4): 431-443, 2019 01 22.
Article
in English
| MEDLINE | ID: mdl-30586695
8.
Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.
N Engl J Med
; 377(9): 819-828, 2017 08 31.
Article
in English
| MEDLINE | ID: mdl-28691885
9.
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9.
N Engl J Med
; 376(1): 41-51, 2017 01 05.
Article
in English
| MEDLINE | ID: mdl-27959715
10.
Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR).
Cardiovasc Drugs Ther
; 34(3): 357-370, 2020 06.
Article
in English
| MEDLINE | ID: mdl-32062791
11.
Correction to: Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR).
Cardiovasc Drugs Ther
; 34(6): 889, 2020 Dec.
Article
in English
| MEDLINE | ID: mdl-32548685
12.
The Nonclinical Safety Profile of GalNAc-conjugated RNAi Therapeutics in Subacute Studies.
Toxicol Pathol
; 46(7): 735-745, 2018 10.
Article
in English
| MEDLINE | ID: mdl-30139307
13.
Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.
Mol Ther
; 25(1): 71-78, 2017 Jan 04.
Article
in English
| MEDLINE | ID: mdl-28129130
14.
Safety and efficacy of RNAi therapy for transthyretin amyloidosis.
N Engl J Med
; 369(9): 819-29, 2013 Aug 29.
Article
in English
| MEDLINE | ID: mdl-23984729
15.
Effect of Preexisting Serum and Mucosal Antibody on Experimental Respiratory Syncytial Virus (RSV) Challenge and Infection of Adults.
J Infect Dis
; 212(11): 1719-25, 2015 Dec 01.
Article
in English
| MEDLINE | ID: mdl-25977264
16.
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.
Lancet
; 383(9911): 60-68, 2014 Jan 04.
Article
in English
| MEDLINE | ID: mdl-24094767
17.
RNA interference in the era of nucleic acid therapeutics.
Nat Biotechnol
; 42(3): 394-405, 2024 Mar.
Article
in English
| MEDLINE | ID: mdl-38409587
18.
Liver as a target for oligonucleotide therapeutics.
J Hepatol
; 59(6): 1354-9, 2013 Dec.
Article
in English
| MEDLINE | ID: mdl-23770039
19.
A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus.
Proc Natl Acad Sci U S A
; 107(19): 8800-5, 2010 May 11.
Article
in English
| MEDLINE | ID: mdl-20421463
20.
RNA interference therapy in lung transplant patients infected with respiratory syncytial virus.
Am J Respir Crit Care Med
; 183(4): 531-8, 2011 Feb 15.
Article
in English
| MEDLINE | ID: mdl-20851929