Timing is everything: Clinical courses of Hunter syndrome associated with age at initiation of therapy in a sibling pair

Hunter syndrome, or mucopolysaccharidosis (MPS) II, is a rare lysosomal disorder characterized by progressive, multi-system disease. As most symptoms cannot be reversed once established, early detection and treatment prior to the onset of clinical symptoms are critical. However, it is difficult to i...

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Detalles Bibliográficos
Autores principales: Grant, Nathan, Sohn, Young Bae, Ellinwood, N. Matthew, Okenfuss, Ericka, Mendelsohn, Bryce A., Lynch, Leslie E., Braunlin, Elizabeth A., Harmatz, Paul R., Eisengart, Julie B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8856919/
https://www.ncbi.nlm.nih.gov/pubmed/35242576
http://dx.doi.org/10.1016/j.ymgmr.2022.100845

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