Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation Results in Patients with Hurler Syndrome: Clinical Cases

Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation Results in Patients with Hurler Syndrome: Clinical Cases

Mucopolysaccharidosis type I (MPS I) is the hereditary disease characterized with alpha-L-iduronidase activity decrease and further accumulation of heparan and dermatan sulfate in lysosomes. MPS I is rare autosomal recessive disorder with incidence of 0.5–4 cases on 100.000 live-birth infants. Meant...

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Bibliographic Details
Main Authors: Nato D. Vashakmadze, Leyla S. Namazova-Baranova, Natalia V. Zhurkova, Ekaterina Yu. Zakharova, Svetlana V. Mikhaylova, Grigory V. Revunenkov
Format: Article
Language:English
Published: "Paediatrician" Publishers LLC 2019-09-01
Series:Вопросы современной педиатрии
Subjects:
children
mucopolysaccharidosis type i
hurler syndrome
transplantation
hematopoietic stem cell
enzyme replacement therapy
survivabilit
Online Access:https://vsp.spr-journal.ru/jour/article/view/2139
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