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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.
Artículo
en Inglés
| MEDLINE | ID: mdl-37940383
2.
Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease.
Genet Med
; 25(12): 100968, 2023 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-37634127
3.
A novel unbiased method reveals progressive podocyte globotriaosylceramide accumulation and loss with age in females with Fabry disease.
Kidney Int
; 102(1): 173-182, 2022 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-35483528
4.
Exponential increase in neutralizing and spike specific antibodies following vaccination of COVID-19 convalescent plasma donors.
Transfusion
; 61(7): 2099-2106, 2021 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-33829513
5.
Initial experience from a renal genetics clinic demonstrates a distinct role in patient management.
Genet Med
; 22(6): 1025-1035, 2020 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-32203225
6.
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.
Artículo
en Inglés
| MEDLINE | ID: mdl-30834538
7.
Correction: Initial experience from a renal genetics clinic demonstrates a distinct role in patient management.
Genet Med
; 23(10): 2017-2019, 2021 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-33024316
8.
The Impact of Fabry Disease on Reproductive Fitness.
JIMD Rep
; 37: 85-97, 2017.
Artículo
en Inglés
| MEDLINE | ID: mdl-28324326
9.
Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease.
Drug Des Devel Ther
; 9: 3435-44, 2015.
Artículo
en Inglés
| MEDLINE | ID: mdl-26185417
10.
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase.
PLoS One
; 10(8): e0134341, 2015.
Artículo
en Inglés
| MEDLINE | ID: mdl-26252393
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