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Journal Abstract Search

281 related items for PubMed ID: 33038326

  • 1. A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II.
    Okuyama T, Eto Y, Sakai N, Nakamura K, Yamamoto T, Yamaoka M, Ikeda T, So S, Tanizawa K, Sonoda H, Sato Y.
    Mol Ther; 2021 Feb 03; 29(2):671-679. PubMed ID: 33038326
    [Abstract] [Full Text] [Related]

  • 2. Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil.
    Giugliani R, Martins AM, So S, Yamamoto T, Yamaoka M, Ikeda T, Tanizawa K, Sonoda H, Schmidt M, Sato Y.
    Mol Ther; 2021 Jul 07; 29(7):2378-2386. PubMed ID: 33781915
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  • 3. Clearance of heparan sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice.
    Morimoto H, Kida S, Yoden E, Kinoshita M, Tanaka N, Yamamoto R, Koshimura Y, Takagi H, Takahashi K, Hirato T, Minami K, Sonoda H.
    Mol Ther; 2021 May 05; 29(5):1853-1861. PubMed ID: 33508431
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  • 4. [Pharmacological property, mechanism of action and clinical study results of Pabinafusp Alfa (Genetical Recombination) (IZCARGO® I.V. Infusion 10 mg) as the therapeutic for Mucopolysaccharidosis type-II (Hunter syndrome)].
    Yamamoto R, Kawashima S.
    Nihon Yakurigaku Zasshi; 2022 May 05; 157(1):62-75. PubMed ID: 34980815
    [Abstract] [Full Text] [Related]

  • 5. Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial.
    Okuyama T, Eto Y, Sakai N, Minami K, Yamamoto T, Sonoda H, Yamaoka M, Tachibana K, Hirato T, Sato Y.
    Mol Ther; 2019 Feb 06; 27(2):456-464. PubMed ID: 30595526
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  • 6. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data.
    Giugliani R, Martins AM, Okuyama T, Eto Y, Sakai N, Nakamura K, Morimoto H, Minami K, Yamamoto T, Yamaoka M, Ikeda T, So S, Tanizawa K, Sonoda H, Schmidt M, Sato Y.
    Int J Mol Sci; 2021 Oct 10; 22(20):. PubMed ID: 34681597
    [Abstract] [Full Text] [Related]

  • 7. Nonclinical safety evaluation of pabinafusp alfa, an anti-human transferrin receptor antibody and iduronate-2-sulfatase fusion protein, for the treatment of neuronopathic mucopolysaccharidosis type II.
    Yamamoto R, Yoden E, Tanaka N, Kinoshita M, Imakiire A, Hirato T, Minami K.
    Mol Genet Metab Rep; 2021 Jun 10; 27():100758. PubMed ID: 33981582
    [Abstract] [Full Text] [Related]

  • 8. Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC/MS/MS.
    Tanaka N, Kida S, Kinoshita M, Morimoto H, Shibasaki T, Tachibana K, Yamamoto R.
    Mol Genet Metab; 2018 Sep 10; 125(1-2):53-58. PubMed ID: 30064964
    [Abstract] [Full Text] [Related]

  • 9. Transferrin Receptor-Targeted Iduronate-2-sulfatase Penetrates the Blood-Retinal Barrier and Improves Retinopathy in Mucopolysaccharidosis II Mice.
    Imakiire A, Morimoto H, Suzuki H, Masuda T, Yoden E, Inoue A, Morioka H, Konaka T, Mori A, Shirasaka R, Kato R, Hirato T, Sonoda H, Minami K.
    Mol Pharm; 2023 Nov 06; 20(11):5901-5909. PubMed ID: 37860991
    [Abstract] [Full Text] [Related]

  • 10. Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan.
    Nakamura K, Sakai N, Hossain MA, Eisengart JB, Yamamoto T, Tanizawa K, So S, Schmidt M, Sato Y.
    Orphanet J Rare Dis; 2024 Mar 07; 19(1):104. PubMed ID: 38454486
    [Abstract] [Full Text] [Related]

  • 11. Divergent developmental trajectories in two siblings with neuropathic mucopolysaccharidosis type II (Hunter syndrome) receiving conventional and novel enzyme replacement therapies: A case report.
    Tomita K, Okamoto S, Seto T, Hamazaki T, So S, Yamamoto T, Tanizawa K, Sonoda H, Sato Y.
    JIMD Rep; 2021 Nov 07; 62(1):9-14. PubMed ID: 34765392
    [Abstract] [Full Text] [Related]

  • 12. Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice.
    Morimoto H, Morioka H, Imakiire A, Yamamoto R, Hirato T, Sonoda H, Minami K.
    Mol Ther Methods Clin Dev; 2022 Jun 09; 25():534-544. PubMed ID: 35662814
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  • 16. Safety and efficacy of enzyme replacement therapy with idursulfase beta in children aged younger than 6 years with Hunter syndrome.
    Sohn YB, Cho SY, Lee J, Kwun Y, Huh R, Jin DK.
    Mol Genet Metab; 2015 Feb 09; 114(2):156-60. PubMed ID: 25219292
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  • 20. The efficacy of intracerebroventricular idursulfase-beta enzyme replacement therapy in mucopolysaccharidosis II murine model: heparan sulfate in cerebrospinal fluid as a clinical biomarker of neuropathology.
    Sohn YB, Ko AR, Seong MR, Lee S, Kim MR, Cho SY, Kim JS, Sakaguchi M, Nakazawa T, Kosuga M, Seo JH, Okuyama T, Jin DK.
    J Inherit Metab Dis; 2018 Nov 09; 41(6):1235-1246. PubMed ID: 29978271
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