Current Approaches to the Treatment of Hunter Syndrome

Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked hereditary disorder associated with a deficiency of iduronate2-sulfatase (IDS). IDS deficiency provokes the accumulation of dermatan sulfate and heparan sulfate in different tissues. Clinical manifestations of MPS II are heteroge...

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Autori principali: Ekaterina Yu. Zakharova, Elena Yu. Voskoboeva, Alla N. Semyachkina, Nato D. Vashakmadze, Amina I. Gamzatova, Svetlana V. Mikhailova, Sergey I. Kutsev
Natura: Artigo
Lingua:Inglês
Pubblicazione: Paediatrician Publishers, LLC 2018-10-01
Serie:Pediatričeskaâ Farmakologiâ
Soggetti:
hunter syndrome
mucopolysaccharidosis type ii
enzyme replacement therapy
gene therapy
hematopoietic stem cell transplantation
Accesso online:https://www.pedpharma.ru/jour/article/view/1653
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