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Hematopoietic Stem Cell Transplantation for Patients with Mucopolysaccharidosis II

30 Jun 2017

Abstract excerpt

There is limited information regarding the long-term outcomes of hematopoietic stem cell transplantation (HSCT) for mucopolysaccharidosis II (MPS II). In this study, clinical, biochemical, and radiologic findings were assessed in patients who underwent HSCT and/or enzyme replacement therapy (ERT). Demographic data for 146 HSCT patients were collected from 27 new cases and 119 published cases and were compared...

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PMID: 28673849
DOI: 10.1016/j.bbmt.2017.06.020

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