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

• Main Authors: Nato V. Vashakmadze (Author), Natalia V. Zhurkova (Author), Marina A. Babaykina (Author), Albina V. Dobrotok (Author), Olga B. Gordeeva (Author), Leyla S. Namazova-Baranova (Author)
• Format: Book
• Published: "Paediatrician" Publishers LLC, 2023-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...