Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter Syndrome)

BACKGROUND: Mucopolysaccharidosis II (MPS II, Hunter syndrome) is a rare X-linked lysosomal storage disorder caused by the deficiency of iduronate-2-sulfatase (IDS). In affected patients, glycosaminoglycan (GAG) accumulates in the lysosomes of many organs and tissues contributing to the pathology as...

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Autores principales: Sohn, Young Bae, Cho, Sung Yoon, Park, Sung Won, Kim, Su Jin, Ko, Ah-Ra, Kwon, Eun-Kyung, Han, Sun Ju, Jin, Dong-Kyu
Formato: Artigo
Lenguaje:Inglês
Publicado: BioMed Central 2013
Materias:
Research
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3614543/
https://ncbi.nlm.nih.gov/pubmed/23497636
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-42
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