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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Bibliografske podrobnosti

Principais autores:	Nato V. Vashakmadze, Natalia V. Zhurkova, Marina A. Babaykina, Albina V. Dobrotok, Olga B. Gordeeva, Leyla S. Namazova-Baranova
Format:	Artigo
Jezik:	Inglês
Izdano:	"Paediatrician" Publishers LLC 2023-12-01
Serija:	Вопросы современной педиатрии
Teme:	mucopolysaccharidosis type i hurler syndrome hematopoietic stem cell transplantation enzyme replacement therapy
Online dostop:	https://vsp.spr-journal.ru/jour/article/view/3361
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