Clinical Outcomes following Hematopoietic Stem Cell Transplantation for the Treatment of Mucopolysaccharidosis VI

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is one of approximately 50 known lysosomal storage disorders. MPS VI is characterized by an absence or deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) resulting in accumulation of dermatan sulfate. Prior to the availability...

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Main Authors: Turbeville, Sean, Nicely, Helen, Rizzo, J. Douglas, Pedersen, Tanya L., Orchard, Paul J., Horwitz, Mitchell E., Horwitz, Edwin M., Veys, Paul, Bonfim, Carmem, Al-Seraihy, Amal
Format: Artigo
Sprog:Inglês
Udgivet: 2010
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3367500/
https://ncbi.nlm.nih.gov/pubmed/20980181
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2010.09.010
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