Combination Treatment for Severe Forms of Mucopolysaccharidosis, Type I (Hurler Syndrome): Case Report

Combination Treatment for Severe Forms of Mucopolysaccharidosis, Type I (Hurler Syndrome): Case Report

Background. Hurler syndrome (mucopolysaccharidosis, type I) is a rare hereditary disease with chronic course. The main methods for Hurler syndrome management are hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT). In recent years, combination treatment (ERT administr...

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Bibliographic Details
Main Authors: Nato V. Vashakmadze, Natalia V. Zhurkova, Marina A. Babaykina, Albina V. Dobrotok, Olga B. Gordeeva, Leyla S. Namazova-Baranova
Format: Article
Language:English
Published: "Paediatrician" Publishers LLC 2023-12-01
Series:Вопросы современной педиатрии
Subjects:
mucopolysaccharidosis
type i
hurler syndrome
hematopoietic stem cell transplantation
enzyme replacement therapy
Online Access:https://vsp.spr-journal.ru/jour/article/view/3361
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https://vsp.spr-journal.ru/jour/article/view/3361

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