Current therapies for Morquio A syndrome and their clinical outcomes.

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第一作者： Kazuki,Sawamoto

第一单位： Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA.

作者： Kazuki,Sawamoto ^[1] ; Yasuyuki,Suzuki ^[2] ; William G,Mackenzie ^[1] ; Mary C,Theroux ^[1] ; Christian,Pizarro ^[1] ; Hiromasa,Yabe ^[3] ; Kenji E,Orii ^[4] ; Robert W,Mason ^[1] ; Tadao,Orii ^[5] ; Shunji,Tomatsu ^[6]

作者单位： Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA. ^[1] Medical Education Development Center, Gifu University, Gifu, Japan. ^[2] Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, Isehara, Japan. ^[3] Division of Neonatal Intensive Care Unit, Gifu University Hospital, Gifu, Japan. ^[4] Department of Pediatrics, Gifu University, Gifu, Japan. ^[5] Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA; Department of Pediatrics, Gifu University, Gifu, Japan. ^[6]

关键词 ERT HSCT Morquio A gene therapy orthopedic surgery tracheal obstruction

DOI 10.1080/21678707.2016.1214572

PMID 28217429

发布时间 2024-03-24

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Current therapies for Morquio A syndrome and their clinical outcomes.

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Current therapies for Morquio A syndrome and their clinical outcomes.

Expert opinion on orphan drugs

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