Hematopoietic cell transplantation for sialidosis type I

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2 Scopus citations


We report the clinical and laboratory follow-up data of an adolescent female with Type I Sialidosis who underwent bone marrow transplant (BMT). After BMT, plasma and urine biomarkers responded concurrently with engraftment. Neuropsychiatry data showed preservation in some domains, but she did have overall decline in motor performance. Sialidosis is a very rare lysosomal condition, and we believe this to be the first report of a case of Type I Sialidosis undergoing BMT.

Original languageEnglish (US)
Article number100832
JournalMolecular Genetics and Metabolism Reports
StatePublished - Mar 2022

Bibliographical note

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  • Blood and marrow transplantation
  • Cherry-red spot myoclonus syndrome
  • Hematopoietic cell transplantation
  • Sialidosis, type I


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