Detalhe da pesquisa
1.
Successful AAV8 readministration: Suppression of capsid-specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease.
J Gene Med
; 25(8): e3509, 2023 08.
Artigo
Inglês
| MEDLINE | ID: mdl-36994804
2.
Transforming the clinical outcome in CRIM-negative infantile Pompe disease identified via newborn screening: the benefits of early treatment with enzyme replacement therapy and immune tolerance induction.
Genet Med
; 23(5): 845-855, 2021 05.
Artigo
Inglês
| MEDLINE | ID: mdl-33495531
3.
The potential impact of timing of IVIG administration on the efficacy of rituximab for immune tolerance induction for patients with Pompe disease.
Clin Immunol
; 219: 108541, 2020 10.
Artigo
Inglês
| MEDLINE | ID: mdl-32681978
4.
An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease.
Genet Med
; 21(4): 887-895, 2019 04.
Artigo
Inglês
| MEDLINE | ID: mdl-30214072
5.
Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation.
Mol Genet Metab
; 123(2): 92-96, 2018 02.
Artigo
Inglês
| MEDLINE | ID: mdl-29289479
6.
Insight into the phenotype of infants with Pompe disease identified by newborn screening with the common c.-32-13T>G "late-onset" GAA variant.
Mol Genet Metab
; 122(3): 99-107, 2017 11.
Artigo
Inglês
| MEDLINE | ID: mdl-28951071
7.
The emerging phenotype of late-onset Pompe disease: A systematic literature review.
Mol Genet Metab
; 120(3): 163-172, 2017 03.
Artigo
Inglês
| MEDLINE | ID: mdl-28185884
8.
An updated management approach of Pompe disease patients with high-sustained anti-rhGAA IgG antibody titers: experience with bortezomib-based immunomodulation.
Front Immunol
; 15: 1360369, 2024.
Artigo
Inglês
| MEDLINE | ID: mdl-38524130
9.
Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy.
Front Immunol
; 15: 1336599, 2024.
Artigo
Inglês
| MEDLINE | ID: mdl-38715621
10.
Development of high sustained anti-drug antibody titers and corresponding clinical decline in a late-onset Pompe disease patient after 11+ years on enzyme replacement therapy.
Mol Genet Metab Rep
; 36: 100981, 2023 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-37342670
11.
Immunophenotype associated with high sustained antibody titers against enzyme replacement therapy in infantile-onset Pompe disease.
Front Immunol
; 14: 1301912, 2023.
Artigo
Inglês
| MEDLINE | ID: mdl-38250073
12.
Outside the fiber: Endomysial stromal and capillary pathology in skeletal muscle may impede infusion therapy in infantile-onset Pompe disease.
J Neuropathol Exp Neurol
; 82(4): 345-362, 2023 03 20.
Artigo
Inglês
| MEDLINE | ID: mdl-36864705
13.
A favorable outcome in an infantile-onset Pompe patient with cross reactive immunological material (CRIM) negative disease with high dose enzyme replacement therapy and adjusted immunomodulation.
Mol Genet Metab Rep
; 32: 100893, 2022 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-35813979
14.
Immune Tolerance-Adjusted Personalized Immunogenicity Prediction for Pompe Disease.
Front Immunol
; 12: 636731, 2021.
Artigo
Inglês
| MEDLINE | ID: mdl-34220802
15.
Benefits of Prophylactic Short-Course Immune Tolerance Induction in Patients With Infantile Pompe Disease: Demonstration of Long-Term Safety and Efficacy in an Expanded Cohort.
Front Immunol
; 11: 1727, 2020.
Artigo
Inglês
| MEDLINE | ID: mdl-32849613
16.
Pulmonary outcome measures in long-term survivors of infantile Pompe disease on enzyme replacement therapy: A case series.
Pediatr Pulmonol
; 55(3): 674-681, 2020 03.
Artigo
Inglês
| MEDLINE | ID: mdl-31899940
17.
A Race Against Time-Changing the Natural History of CRIM Negative Infantile Pompe Disease.
Front Immunol
; 11: 1929, 2020.
Artigo
Inglês
| MEDLINE | ID: mdl-33013846
18.
Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy.
Mol Genet Metab Rep
; 20: 100475, 2019 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-31193175
19.
Immunological challenges and approaches to immunomodulation in Pompe disease: a literature review.
Ann Transl Med
; 7(13): 285, 2019 Jul.
Artigo
Inglês
| MEDLINE | ID: mdl-31392197
20.
Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease.
JCI Insight
; 2(16)2017 Aug 17.
Artigo
Inglês
| MEDLINE | ID: mdl-28814660