Enzyme replacement therapy in Hurler syndrome after failure of hematopoietic transplant

Molecular Genetics and Metabolism Reports

Leonor Arranz¹  Luis Aldamiz-Echevarria² 

[1] Pediatrics Department, Donostia Universitary Hospital, San Sebastian, Spain;Pediatrics Department, Faculty of Medicine, Basque Country University (UPV/EHU), San Sebastian, Spain;

关键词: Hurler syndrome;    Enzyme replacement therapy;    Hematopoietic transplant;    Mucopolysaccharosidosis type I;    Laronidase;    Anti-laronidase antibodies;   

DOI  :  10.1016/j.ymgmr.2015.04.003

来源: DOAJ

【 授权许可】

Unknown