Efficacy of early haematopoietic stem cell transplantation versus enzyme replacement therapy on neurological progression in severe Hunter syndrome: Case report of siblings and literature review

Hunter syndrome is a neurodegenerative lysosomal storage disorder with limited treatment options to halt the progressive neurocognitive decline. Whilst Intravenous enzyme replacement therapy (ERT) does not cross the blood brain barrier; Intrathecal ERT, in clinical studies, did not demonstrate signi...

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Detalles Bibliográficos
Autores principales: Sreekantam, Srividya, Smith, Laura, Stewart, Catherine, Kearney, Shauna, Lawson, Sarah, Raiman, Julian, Vijay, Suresh, Santra, Saikat
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9160838/
https://www.ncbi.nlm.nih.gov/pubmed/35663302
http://dx.doi.org/10.1016/j.ymgmr.2022.100881

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