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

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

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’ qual...

Full description

Bibliographic Details
Main Authors: Natalia V. Zhurkova, Nato D. Vashakmadze, Ludmila K. Mikhaylova, Marina A. Babaykina, Nina V. Fedorova, Elena Yu. Voskoboeva, Ekaterina Yu. Zakharova, Leyla S. Namazova-Baranova
Format: Article
Language:English
Published: "Paediatrician" Publishers LLC 2023-01-01
Series:Вопросы современной педиатрии
Subjects:
мукополисахаридоз
тип ii
ферментозаместительная терапия
гликозаминогликаны
Online Access:https://vsp.spr-journal.ru/jour/article/view/3072

Similar Items

Similar Items