Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series

Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series

Abstract Background Multiple sulfatase deficiency (MSD) is an exceptionally rare neurodegenerative disorder due to the absence or deficiency of 17 known cellular sulfatases. The activation of all these cellular sulfatases is dependent on the presence of the formylglycine-generating enzyme, which is...

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Main Authors: Nishitha R. Pillai, Ning Liu, Xiyuan Li, Xiqi Li, Rebecca Ahrens-Nicklas, Laura Adang, Julie B. Eisengart, Grace Bronken, Ashish Gupta, Troy C. Lund, Chester B. Whitley, Sarah H. Elsea, Paul J. Orchard
Format: Article
Language:English
Published: Nature Portfolio 2025-01-01
Series:Communications Medicine
Online Access:https://doi.org/10.1038/s43856-024-00703-8
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