Mucopolysaccharidosis type II: Enzyme Replacement Therapy Efficiency

Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is the hereditary lysosomal storage disease caused by pathological variants in IDS gene. Such variants lead to iduronate-2-sulfatase enzyme deficiency and glycosaminoglycan catabolism disorder. Major clinical signs are central nervous syste...

書誌詳細
出版年:	Вопросы современной педиатрии
主要な著者:	Nato D. Vashakmadze, Leyla S. Namazova-Baranova, Natalia V. Zhurkova, Ekaterina Yu. Zakharova, Grigory V. Revunenkov, Tina V. Lobjanidze, Marina A. Babaikina
フォーマット:	論文
言語:	英語
出版事項:	"Paediatrician" Publishers LLC 2020-02-01
主題:	children lysosomal diseases mucopolysaccharidosis type ii cardiovascular system enzyme replacement therapy idursulfase
オンライン･アクセス:	https://vsp.spr-journal.ru/jour/article/view/2264

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