Detalhe da pesquisa
1.
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.
N Engl J Med
; 375(6): 545-55, 2016 Aug 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-27509102
2.
Comments on: Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice.
J Pharmacol Exp Ther
; 389(3): 310-312, 2024 May 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-38772713
3.
The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat.
Genet Med
; 19(4): 430-438, 2017 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-27657681
4.
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
J Neurol
; 271(5): 2810-2823, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38418563
5.
Therapeutic Role of Pharmacological Chaperones in Lysosomal Storage Disorders: A Review of the Evidence and Informed Approach to Reclassification.
Biomolecules
; 13(8)2023 08 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-37627292
6.
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
Lancet Neurol
; 20(12): 1027-1037, 2021 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-34800400
7.
Novel quantitative method to evaluate globotriaosylceramide inclusions in renal peritubular capillaries by virtual microscopy in patients with fabry disease.
Arch Pathol Lab Med
; 136(7): 816-24, 2012 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-22742555
8.
Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies.
Orphanet J Rare Dis
; 7: 91, 2012 Nov 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-23176611