Detalhe da pesquisa
1.
Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.
J Med Genet
; 2023 Nov 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37940383
2.
Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial.
J Inherit Metab Dis
; 42(3): 534-544, 2019 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-30834538
3.
Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.
Am J Hematol
; 91(7): 661-5, 2016 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-27102949
4.
Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naïve patients with Gaucher disease.
Am J Hematol
; 91(7): 656-60, 2016 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-27174694
5.
Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease.
Blood Cells Mol Dis
; 54(1): 9-16, 2015 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-25453586
6.
Preclinical and first-in-human evaluation of PRX-105, a PEGylated, plant-derived, recombinant human acetylcholinesterase-R.
Toxicol Appl Pharmacol
; 287(3): 202-9, 2015 Sep 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26051873
7.
A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase.
Blood Cells Mol Dis
; 53(4): 253-60, 2014 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-24950666
8.
Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease.
Blood
; 118(22): 5767-73, 2011 Nov 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-21900191
9.
Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study.
Orphanet J Rare Dis
; 18(1): 332, 2023 Oct 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-37865771
10.
Improving the accuracy of MRI spleen and liver volume measurements: a phase III Gaucher disease clinical trial setting as a model.
Blood Cells Mol Dis
; 46(1): 47-52, 2011 Jan 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-21084202
11.
Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system.
Plant Biotechnol J
; 5(5): 579-90, 2007 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-17524049
12.
Immunogenicity of glycans on biotherapeutic drugs produced in plant expression systems-The taliglucerase alfa story.
PLoS One
; 12(10): e0186211, 2017.
Artigo
em Inglês
| MEDLINE | ID: mdl-29088235
13.
A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation.
PLoS One
; 4(3): e4792, 2009.
Artigo
em Inglês
| MEDLINE | ID: mdl-19277123
14.
Ex vivo transduction of human dermal tissue structures for autologous implantation production and delivery of therapeutic proteins.
Mol Ther
; 12(2): 274-82, 2005 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-16043098
15.
Human erythropoietin gene therapy for patients with chronic renal failure.
Blood
; 106(7): 2280-6, 2005 Oct 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-15798000