Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA

Molecular Genetics and Metabolism Reports

Nobuyuki Hyakuna¹  Yasutsugu Chinen¹  Tadao Orii²  Takeshi Higa³  Yasuyuki Suzuki⁴  Shunji Tomatsu⁵ 

[1] Department of Pediatrics, Faculty of Medicine, University of the Ryukyus, Japan;Department of Pediatrics, Gifu University, Gifu, Japan;Department of Pediatrics, Okinawa Prefectural Nanbu Medical Center Children's Medical Center, Japan;Medical Education Development Center, Gifu University, Japan;Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA;

关键词: Mucopolysaccharidosis IVA;    Bone marrow transplantation;    GALNS;    Respiratory function;    Bone mineral density;   

DOI  :  10.1016/j.ymgmr.2013.11.002

来源: DOAJ

【 授权许可】

Unknown