Impact of the Timing of Enzyme Replacement Therapy Initiation and Cognitive Impairment Status on Outcomes for Patients with Mucopolysaccharidosis II (MPS II) in the United States: A Retrospective Chart Review

Background: Mucopolysaccharidosis II (MPS II; Hunter syndrome; OMIM 309900) is a rare, X-linked, lysosomal storage disease caused by deficient iduronate-2-sulfatase activity. Accumulation of glycosaminoglycans results in multisystemic disease manifestations, which may include central nervous system...

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Detalles Bibliográficos
Autores principales: Yee, Karen S., Alexanderian, David, Feng, Yidie, Ren, Xiaowei, Schweikert, Bernd, Ayodele, Olulade
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Columbia Data Analytics, LLC 2022
Materias:
Neurological Diseases
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9424538/
https://www.ncbi.nlm.nih.gov/pubmed/36168594
http://dx.doi.org/10.36469/001c.36540

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9424538/
https://www.ncbi.nlm.nih.gov/pubmed/36168594
http://dx.doi.org/10.36469/001c.36540

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