Detalhe da pesquisa
1.
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial.
Mol Ther
; 32(3): 609-618, 2024 Mar 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-38204164
2.
Transferrin Receptor-Targeted Iduronate-2-sulfatase Penetrates the Blood-Retinal Barrier and Improves Retinopathy in Mucopolysaccharidosis II Mice.
Mol Pharm
; 20(11): 5901-5909, 2023 11 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37860991
3.
Calcium sensing and signaling are impaired in the lumbar spine of a rat model of congenital kyphosis.
Eur Spine J
; 32(10): 3403-3412, 2023 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-37555955
4.
Trends in cervical laminoplasty and 30-day postoperative complications: 10-year results from a retrospective, multi-institutional study of 1095 patients.
Eur Spine J
; 32(10): 3575-3582, 2023 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-37624437
5.
Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil.
Mol Ther
; 29(7): 2378-2386, 2021 07 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33781915
6.
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II.
Mol Ther
; 29(2): 671-679, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33038326
7.
Clearance of heparan sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice.
Mol Ther
; 29(5): 1853-1861, 2021 05 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33508431
8.
Drug delivery for neuronopathic lysosomal storage diseases: evolving roles of the blood brain barrier and cerebrospinal fluid.
Metab Brain Dis
; 37(6): 1745-1756, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35088290
9.
Pathogenic Roles of Heparan Sulfate and Its Use as a Biomarker in Mucopolysaccharidoses.
Int J Mol Sci
; 23(19)2022 Oct 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36233030
10.
Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data.
Int J Mol Sci
; 22(20)2021 Oct 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34681597
11.
Upregulated miR-224-5p suppresses osteoblast differentiation by increasing the expression of Pai-1 in the lumbar spine of a rat model of congenital kyphoscoliosis.
Mol Cell Biochem
; 475(1-2): 53-62, 2020 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-32748313
12.
Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial.
Mol Ther
; 27(2): 456-464, 2019 02 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-30595526
13.
Recurrent lumbar-origin osteoblastoma treated with multiple surgery and carbon ion radiotherapy: a case report.
BMC Musculoskelet Disord
; 21(1): 321, 2020 May 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-32443969
14.
The retinol-retinoic acid metabolic pathway is impaired in the lumbar spine of a rat model of congenital kyphoscoliosis.
J Cell Biochem
; 120(9): 15007-15017, 2019 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-31056772
15.
A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II.
Mol Ther
; 26(5): 1366-1374, 2018 05 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29606503
16.
Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice.
Mol Ther Methods Clin Dev
; 29: 439-449, 2023 Jun 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37251981
17.
Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice.
Mol Ther Methods Clin Dev
; 25: 534-544, 2022 Jun 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35662814
18.
Treatment of Neuronopathic Mucopolysaccharidoses with Blood-Brain Barrier-Crossing Enzymes: Clinical Application of Receptor-Mediated Transcytosis.
Pharmaceutics
; 14(6)2022 Jun 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35745811
19.
Treatment of CLN1 disease with a blood-brain barrier penetrating lysosomal enzyme.
Mol Genet Metab Rep
; 33: 100930, 2022 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-36324638
20.
Pharmacokinetics and pharmacodynamics of once-weekly administration of JR-142, a long-acting albumin-fused human growth hormone: A rondemized, placebo-controlled phase 1 study.
Growth Horm IGF Res
; 67: 101500, 2022 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-36113378