α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial Article Swipe

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Paul Harmatz , Roberto Giugliani , Ana Maria Martins , Takashi Hamazaki , Toru Kubo , Ryutaro Kira , Kohtaro Minami , Toshiaki Ikeda , Hiroaki Moriuchi , Satoshi Kawashima , Naoko Takasao , Sairei So , Hiroyuki Sonoda , Tohru Hirato , Kazunori Tanizawa , Mathias Schmidt , Yuji Sato ·

YOU? · · 2024 · Open Access · · DOI: https://doi.org/10.1016/j.ymthe.2024.01.009

Related Topics

Hematopoietic Stem Cell Transplantation

Concepts

Hurler syndrome Mucopolysaccharidosis I Enzyme replacement therapy Transferrin receptor Heparan sulfate Mucopolysaccharidosis type I Dermatan sulfate Mucopolysaccharidosis type II Medicine Pharmacokinetics Pharmacology Transplantation Hematopoietic stem cell transplantation Antibody Immunology Transferrin Internal medicine Disease Heparin

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Type: article
Language: en
Landing Page: https://doi.org/10.1016/j.ymthe.2024.01.009
PDF: http://www.cell.com/article/S1525001624000091/pdf
OA Status: hybrid
Cited By: 9
References: 16
Related Works: 10
OpenAlex ID: https://openalex.org/W4390743640

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OpenAlex ID: https://openalex.org/W4390743640

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DOI: https://doi.org/10.1016/j.ymthe.2024.01.009

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Title: α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial

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Type: article

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Language: en

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Publication year: 2024

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Publication date: 2024-01-09

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Authors: Paul Harmatz, Roberto Giugliani, Ana Maria Martins, Takashi Hamazaki, Toru Kubo, Ryutaro Kira, Kohtaro Minami, Toshiaki Ikeda, Hiroaki Moriuchi, Satoshi Kawashima, Naoko Takasao, Sairei So, Hiroyuki Sonoda, Tohru Hirato, Kazunori Tanizawa, Mathias Schmidt, Yuji Sato

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Landing page: https://doi.org/10.1016/j.ymthe.2024.01.009

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PDF URL: https://www.cell.com/article/S1525001624000091/pdf

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OA status: hybrid

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OA URL: https://www.cell.com/article/S1525001624000091/pdf

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Concepts: Hurler syndrome, Mucopolysaccharidosis I, Enzyme replacement therapy, Transferrin receptor, Heparan sulfate, Mucopolysaccharidosis type I, Dermatan sulfate, Mucopolysaccharidosis type II, Medicine, Pharmacokinetics, Pharmacology, Transplantation, Hematopoietic stem cell transplantation, Antibody, Immunology, Transferrin, Internal medicine, Disease, Heparin

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Cited by: 9

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Citations by year (recent): 2025: 7, 2024: 2

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References (count): 16

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Related works (count): 10

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