Results of 14-year-long Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II: Clinical Case

• Main Authors: Natalia V. Zhurkova (Author), Nato D. Vashakmadze (Author), Ludmila K. Mikhaylova (Author), Marina A. Babaykina (Author), Nina V. Fedorova (Author), Elena Yu. Voskoboeva (Author), Ekaterina Yu. Zakharova (Author), Leyla S. Namazova-Baranova (Author)
• Format: Book
• Published: "Paediatrician" Publishers LLC, 2023-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Background. Mucopolysaccharidosis type II (MPS II) is a rare hereditary disease from the group of lysosomal storage diseases, with progressive course. There is effective enzyme replacement therapy (ERT) for this disease, it prevents the development of severe complications and improves patients'...