Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease

BACKGROUND: Mucopolysaccharidosis type I (MPS I) is a debilitating hereditary disease characterized by alpha-L-iduronidase (IDUA) deficiency and consequent inability to degrade glycosaminoglycans. The pathological accumulation of glycosaminoglycans systemically results in severe mental retardation a...

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Detaylı Bibliyografya
Yayımlandı:Orphanet J Rare Dis
Asıl Yazarlar: Hsu, Yu-Hone, Liu, Ren-Shyan, Lin, Win-Li, Yuh, Yeong-Seng, Lin, Shuan-Pei, Wong, Tai-Tong
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: BioMed Central 2017
Konular:
Research
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC5465581/
https://ncbi.nlm.nih.gov/pubmed/28595620
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0649-6
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