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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2022
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Materias: | |
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 |