Enzyme Replacement Therapy with Idursulfase in Patients with Mucopolysaccharidosis Type II: Literature Review

Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is X-linked hereditary disease from the group of lysosomal storage disease. Its prevalence is 3–7 cases per 1 million live-born boys. MPS II occurs due to the deficiency of iduronate-2-sulfatase enzyme because of pathological changes in the str...

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Autors principals: Nato D. Vashakmadze, Natalya V. Zhurkova, Olga B. Gordeeva, Elena V. Komarova, Tatyana E. Privalova, Anastasya I. Rykunova, Marina А. Babaykina
Format: Artigo
Idioma:Inglês
Publicat: "Paediatrician" Publishers LLC 2021-12-01
Col·lecció:Voprosy Sovremennoj Pediatrii
Matèries:
mucopolysaccharidosis type ii
hunter syndrome
idursulfase
enzyme replacement therapy
survivability
Accés en línia:https://vsp.spr-journal.ru/jour/article/view/2791
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