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Hematopoietic cell transplantation for sialidosis type I

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 o...

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Detalles Bibliográficos
Autores principales: Gupta, Ashish O., Patterson, Marc C., Wood, Tim, Eisengart, Julie B., Orchard, Paul J., Lund, Troy C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8856904/
https://www.ncbi.nlm.nih.gov/pubmed/35242566
http://dx.doi.org/10.1016/j.ymgmr.2021.100832