Detalhe da pesquisa
1.
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.
JAMA
; 330(15): 1448-1458, 2023 10 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-37768671
2.
Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome.
N Engl J Med
; 381(6): 531-542, 2019 08 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-31390500
3.
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.
N Engl J Med
; 379(1): 22-31, 2018 Jul 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29972757
4.
Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides.
N Engl J Med
; 377(3): 222-232, 2017 07 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-28538111
5.
N-acetyl galactosamine-conjugated antisense drug to APOC3 mRNA, triglycerides and atherogenic lipoprotein levels.
Eur Heart J
; 40(33): 2785-2796, 2019 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31329855
6.
Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia.
N Engl J Med
; 373(5): 438-47, 2015 Jul 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-26222559
7.
Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomised, double-blind, placebo-controlled, dose-ranging trials.
Lancet
; 388(10057): 2239-2253, 2016 11 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-27665230
8.
Targeting APOC3 in the familial chylomicronemia syndrome.
N Engl J Med
; 371(23): 2200-6, 2014 Dec 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-25470695
9.
Assessing mNIS+7Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial.
Muscle Nerve
; 56(5): 901-911, 2017 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-28063170
10.
Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study.
Lancet
; 386(10002): 1472-83, 2015 Oct 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-26210642
11.
A randomized, open-label, multicentre, phase 2/3 study to evaluate the safety and efficacy of lumiliximab in combination with fludarabine, cyclophosphamide and rituximab versus fludarabine, cyclophosphamide and rituximab alone in subjects with relapsed chronic lymphocytic leukaemia.
Br J Haematol
; 167(4): 466-77, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-25130401
12.
Safety, immunogenicity and efficacy of the self-amplifying mRNA ARCT-154 COVID-19 vaccine: pooled phase 1, 2, 3a and 3b randomized, controlled trials.
Nat Commun
; 15(1): 4081, 2024 May 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38744844
13.
A phase I/II randomized, double-blinded, placebo-controlled trial of a self-amplifying Covid-19 mRNA vaccine.
NPJ Vaccines
; 7(1): 161, 2022 Dec 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-36513697
14.
Immune gene expression analysis indicates the potential of a self-amplifying Covid-19 mRNA vaccine.
NPJ Vaccines
; 7(1): 154, 2022 Nov 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-36443317
15.
Underlying Immune Disorder May Predispose Some Transthyretin Amyloidosis Subjects to Inotersen-Mediated Thrombocytopenia.
Nucleic Acid Ther
; 30(2): 94-103, 2020 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32043907
16.
Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis.
J Neurol
; 267(4): 1070-1079, 2020 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-31853709
17.
An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials.
CNS Drugs
; 33(9): 919-932, 2019 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-31420846
18.
IONIS-PKKRx a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production.
Nucleic Acid Ther
; 29(2): 82-91, 2019 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-30817230
19.
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.
J Clin Lipidol
; 12(1): 122-129.e2, 2018.
Artigo
em Inglês
| MEDLINE | ID: mdl-29174389
20.
Kind and distribution of cutaneous sensation loss in hereditary transthyretin amyloidosis with polyneuropathy.
J Neurol Sci
; 394: 78-83, 2018 11 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-30219500