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Title:

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.

Document type:
Journal Article; Review
Author(s):
Peters, C; Steward, CG
Abstract:
For the past two decades, hematopoietic cell transplantation (HCT) has been used as effective therapy for selected inherited metabolic diseases (IMD) including Hurler (MPS IH) and Maroteaux-Lamy (MPS VI) syndromes, childhood-onset cerebral X-linked adrenoleukodystrophy (X-ALD), globoid-cell leukodystrophy (GLD), metachromatic leukodystrophy (MLD), alpha-mannosidosis, osteopetrosis, and others. Careful pre-HCT evaluation is critical and coordinated, multidisciplinary follow-up is essential in thi...     »
Journal title abbreviation:
Bone Marrow Transplant
Year:
2003
Journal volume:
31
Journal issue:
4
Pages contribution:
229-39
Language:
eng
Fulltext / DOI:
doi:10.1038/sj.bmt.1703839
Pubmed ID:
http://view.ncbi.nlm.nih.gov/pubmed/12621457
Print-ISSN:
0268-3369
TUM Institution:
Klinik und Poliklinik für Kinderheilkunde und Jugendmedizin
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