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Enzyme replacement therapy in newborn mucopolysaccharidosis IVA mice: early treatment rescues bone lesions?
Tomatsu, Shunji; Montaño, Adriana M; Oikawa, Hirotaka; Dung, Vu Chi; Hashimoto, Amiko; Oguma, Toshihiro; Gutiérrez, Monica L; Takahashi, Tatsuo; Shimada, Tsutomu; Orii, Tadao; Sly, William S.
Affiliation
  • Tomatsu S; Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA. Electronic address: stomatsu@nemours.org.
  • Montaño AM; Department of Pediatrics, Saint Louis University Doisy Research Center, St. Louis, MO, USA; Department of Biochemistry and Molecular Biology, Saint Louis University School of Medicine, St. Louis, MO, USA.
  • Oikawa H; Suzuka University of Medical Science, School of Pharmacy, Japan.
  • Dung VC; Department of Endocrinology, Metabolism & Genetics, Vietnam National Hospital of Pediatrics, Hanoi, Viet Nam.
  • Hashimoto A; Myojinshita Aoki Dermatology Clinic, Tokyo, Japan.
  • Oguma T; Daiichi-Sankyo Pharmaceutical Co., Tokyo, Japan.
  • Gutiérrez ML; Department of Pediatrics, Saint Louis University Doisy Research Center, St. Louis, MO, USA.
  • Takahashi T; Department of Clinical Pharmacy, Faculty of Pharmaceutical Sciences, Hokuriku University, Kanazawa, Japan.
  • Shimada T; Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA.
  • Orii T; Department of Pediatrics, Gifu University, School of Medicine, Gifu, Japan.
  • Sly WS; Department of Biochemistry and Molecular Biology, Saint Louis University School of Medicine, St. Louis, MO, USA.
Mol Genet Metab ; 114(2): 195-202, 2015 Feb.
Article in En | MEDLINE | ID: mdl-24953405
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Bone Diseases / Chondroitinsulfatases / Mucopolysaccharidosis IV / Enzyme Replacement Therapy Limits: Animals Language: En Journal: Mol Genet Metab Year: 2015 Document type: Article
Fulltext
Print
XML
PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Bone Diseases / Chondroitinsulfatases / Mucopolysaccharidosis IV / Enzyme Replacement Therapy Limits: Animals Language: En Journal: Mol Genet Metab Year: 2015 Document type: Article