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1.
Gene Therapy in Combination with Nitrogen Scavenger Pretreatment Corrects Biochemical and Behavioral Abnormalities of Infant Citrullinemia Type 1 Mice.
Int J Mol Sci
; 23(23)2022 Nov 29.
Artículo
en Inglés
| MEDLINE | ID: mdl-36499263
2.
Liver Expression of a MiniATP7B Gene Results in Long-Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice.
Hepatology
; 70(1): 108-126, 2019 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-30706949
3.
Low-dose agalsidase beta treatment in male pediatric patients with Fabry disease: A 5-year randomized controlled trial.
Mol Genet Metab
; 127(1): 86-94, 2019 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-30987917
4.
Effective clearance of GL-3 in a human iPSC-derived cardiomyocyte model of Fabry disease.
J Inherit Metab Dis
; 37(6): 1013-22, 2014 Nov.
Artículo
en Inglés
| MEDLINE | ID: mdl-24850378
5.
Characteristics of patients with Wilson disease in the United States: An insurance claims database study.
World J Hepatol
; 16(5): 791-799, 2024 May 27.
Artículo
en Inglés
| MEDLINE | ID: mdl-38818282
6.
Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy.
JHEP Rep
; 5(10): 100844, 2023 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-37701337
7.
Epidemiology, treatment and burden of Wilson disease in France: A 10-year analysis of the national health insurance database.
Clin Res Hepatol Gastroenterol
; 46(10): 101992, 2022 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-35793759
8.
High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease.
Mol Ther Methods Clin Dev
; 26: 98-106, 2022 Sep 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-35795774
9.
Optimising the IgG-degrading enzyme treatment regimen for enhanced adeno-associated virus transduction in the presence of neutralising antibodies.
Clin Transl Immunology
; 11(2): e1375, 2022.
Artículo
en Inglés
| MEDLINE | ID: mdl-35228870
10.
Evaluation of a low dose, after a standard therapeutic dose, of agalsidase beta during enzyme replacement therapy in patients with Fabry disease.
Genet Med
; 11(4): 256-64, 2009 Apr.
Artículo
en Inglés
| MEDLINE | ID: mdl-19265719
11.
A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease.
Mol Genet Metab
; 96(1): 4-12, 2009 Jan.
Artículo
en Inglés
| MEDLINE | ID: mdl-19022694
12.
Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model.
Nat Commun
; 10(1): 5694, 2019 12 13.
Artículo
en Inglés
| MEDLINE | ID: mdl-31836711
13.
Characterization of early disease status in treatment-naive male paediatric patients with Fabry disease enrolled in a randomized clinical trial.
PLoS One
; 10(5): e0124987, 2015.
Artículo
en Inglés
| MEDLINE | ID: mdl-25955246
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