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Journal Abstract Search

164 related items for PubMed ID: 35662814

  • 1. 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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  • 2. 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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  • 3. [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
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  • 4. 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 05; 27():100758. PubMed ID: 33981582
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  • 5. 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
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  • 7. 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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  • 8. 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 07; 41(6):1235-1246. PubMed ID: 29978271
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  • 10. 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
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  • 11. 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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  • 12. 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 06; 62(1):9-14. PubMed ID: 34765392
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  • 16. A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II.
    Sonoda H, Morimoto H, Yoden E, Koshimura Y, Kinoshita M, Golovina G, Takagi H, Yamamoto R, Minami K, Mizoguchi A, Tachibana K, Hirato T, Takahashi K.
    Mol Ther; 2018 May 02; 26(5):1366-1374. PubMed ID: 29606503
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  • 17. Differences in MPS I and MPS II Disease Manifestations.
    Hampe CS, Yund BD, Orchard PJ, Lund TC, Wesley J, McIvor RS.
    Int J Mol Sci; 2021 Jul 23; 22(15):. PubMed ID: 34360653
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  • 18. α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial.
    Harmatz P, Giugliani R, Martins AM, Hamazaki T, Kubo T, Kira R, Minami K, Ikeda T, Moriuchi H, Kawashima S, Takasao N, So S, Sonoda H, Hirato T, Tanizawa K, Schmidt M, Sato Y.
    Mol Ther; 2024 Mar 06; 32(3):609-618. PubMed ID: 38204164
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  • 19. Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.
    Arguello A, Meisner R, Thomsen ER, Nguyen HN, Ravi R, Simms J, Lo I, Speckart J, Holtzman J, Gill TM, Chan D, Cheng Y, Chiu CL, Dugas JC, Fang M, Lopez IA, Solanoy H, Tsogtbaatar B, Zhu Y, Bhalla A, Henne KR, Henry AG, Delucchi A, Costanzo S, Harris JM, Diaz D, Scearce-Levie K, Sanchez PE.
    JCI Insight; 2021 Oct 08; 6(19):. PubMed ID: 34622797
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  • 20. Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer.
    Laoharawee K, Podetz-Pedersen KM, Nguyen TT, Evenstar LB, Kitto KF, Nan Z, Fairbanks CA, Low WC, Kozarsky KF, McIvor RS.
    Hum Gene Ther; 2017 Aug 08; 28(8):626-638. PubMed ID: 28478695
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