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

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

Abstract 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 retar...

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Bibliographic Details
Main Authors: Yu-Hone Hsu, Ren-Shyan Liu, Win-Li Lin, Yeong-Seng Yuh, Shuan-Pei Lin, Tai-Tong Wong
Format: Article
Language:English
Published: BMC 2017-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Mucopolysaccharidosis type I
Blood-brain barrier
Ultrasound
Recombinant human alpha-L-iduronidase
Online Access:http://link.springer.com/article/10.1186/s13023-017-0649-6

Internet

http://link.springer.com/article/10.1186/s13023-017-0649-6

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