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Journal Abstract Search

173 related items for PubMed ID: 38454486

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  • 3. 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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  • 4. 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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  • 5. Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II.
    Yee KS, Lewis S, Evans E, Romano C, Alexanderian D.
    Orphanet J Rare Dis; 2024 Mar 10; 19(1):110. PubMed ID: 38462612
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  • 6. 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. 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 03; 27():100758. PubMed ID: 33981582
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  • 8. 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
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  • 9. Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS).
    Muenzer J, Botha J, Harmatz P, Giugliani R, Kampmann C, Burton BK.
    Orphanet J Rare Dis; 2021 Oct 30; 16(1):456. PubMed ID: 34717704
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  • 10. 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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  • 11. Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome).
    da Silva EM, Strufaldi MW, Andriolo RB, Silva LA.
    Cochrane Database Syst Rev; 2014 Jan 08; (1):CD008185. PubMed ID: 24399699
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  • 12. Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome).
    da Silva EM, Strufaldi MW, Andriolo RB, Silva LA.
    Cochrane Database Syst Rev; 2011 Nov 09; (11):CD008185. PubMed ID: 22071845
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  • 13. Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future.
    Whiteman DA, Kimura A.
    Drug Des Devel Ther; 2017 Nov 09; 11():2467-2480. PubMed ID: 28860717
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  • 14. Safety and efficacy of idursulfase in the treatment of mucopolysaccharidosis II (Hunter syndrome): a post-marketing study in Japan.
    Ueda K, Hokugo J.
    Expert Opin Drug Saf; 2020 Jul 09; 19(7):891-901. PubMed ID: 32342708
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  • 15. Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series.
    Lampe C, Bosserhoff AK, Burton BK, Giugliani R, de Souza CF, Bittar C, Muschol N, Olson R, Mendelsohn NJ.
    J Inherit Metab Dis; 2014 Sep 09; 37(5):823-9. PubMed ID: 24596019
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  • 16. 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 09; 62(1):9-14. PubMed ID: 34765392
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  • 17. Japan Elaprase Treatment (JET) study: idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II).
    Okuyama T, Tanaka A, Suzuki Y, Ida H, Tanaka T, Cox GF, Eto Y, Orii T.
    Mol Genet Metab; 2010 Jan 09; 99(1):18-25. PubMed ID: 19773189
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  • 18. Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS).
    Muenzer J, Giugliani R, Scarpa M, Tylki-Szymańska A, Jego V, Beck M.
    Orphanet J Rare Dis; 2017 Oct 03; 12(1):161. PubMed ID: 28974237
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  • 19. Diagnosis, quality of life, and treatment of patients with Hunter syndrome in the French healthcare system: a retrospective observational study.
    Guffon N, Heron B, Chabrol B, Feillet F, Montauban V, Valayannopoulos V.
    Orphanet J Rare Dis; 2015 Apr 12; 10():43. PubMed ID: 25887606
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  • 20. Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome).
    Sohn YB, Cho SY, Park SW, Kim SJ, Ko AR, Kwon EK, Han SJ, Jin DK.
    Orphanet J Rare Dis; 2013 Mar 18; 8():42. PubMed ID: 23497636
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