Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series
These data suggest biochemical benefits post-transplant along with slowing of disease progression. Long-term follow-up is necessary to fully evaluate the therapeutic benefit of HCT in MSD.
Authors
- Laura Adang (ORCID: https://orcid.org/0000-0002-8454-1116)
- Sarah H. Elsea (ORCID: https://orcid.org/0000-0002-1400-8519)
- Julie B. Eisengart (ORCID: https://orcid.org/0000-0001-5340-017X)
- Nishitha R. Pillai (ORCID: https://orcid.org/0000-0002-2943-2136)
- Grace Bronken (ORCID: https://orcid.org/0000-0001-5316-0882)
- Troy C. Lund (ORCID: https://orcid.org/0000-0001-9174-8919)
- Xiyuan Li (ORCID: https://orcid.org/0000-0003-3192-652X)
- Paul J. Orchard (ORCID: https://orcid.org/0000-0001-9426-1292)
- Xiqi Li
- Ashish Gupta (ORCID: https://orcid.org/0000-0001-8334-8552)
- Rebecca C. Ahrens‐Nicklas (ORCID: https://orcid.org/0000-0001-8243-7123)
- Ning Liu (ORCID: https://orcid.org/0000-0002-9487-9305)
- Chester B. Whitley (ORCID: https://orcid.org/0000-0001-8895-6485)
Institutions
- University of Minnesota (US)
- Fairview Health Services (US)
- Children's Hospital of Philadelphia (US)
- Baylor College of Medicine (US)
- Baylor Genetics (US)
Publication Details
- Journal
- Communications Medicine
- Published
- 2025-01-09
- DOI
- https://doi.org/10.1038/s43856-024-00703-8
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16