Advancements in the Treatment of Mucopolysaccharidoses: From Established Therapies to Gene Therapy

Advancements in the Treatment of Mucopolysaccharidoses: From Established Therapies to Gene Therapy

Introduction and purpose Mucopolysaccharidoses (MPS), a subset of inborn errors of metabolism (IEM), are genetic disorders requiring pediatricians to recognize non-specific symptoms and carefully monitor newborns. Early diagnosis is essential for optimizing therapeutic outcomes. In recent years, s...

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Bibliographic Details
Main Authors: Joanna Wanat, Wojciech Homa, Aleksandra Warunek, Gabriela Gronowicz, Izabela Dzikowska, Agata Siejka, Daria Stefaniak, Weronika Zielińska, Michał Chról
Format: Article
Language:English
Published: Kazimierz Wielki University 2025-02-01
Series:Journal of Education, Health and Sport
Subjects:
Gene therapy
Mucopolysaccharidoses
Hurler syndrome
Hematopoietic stem cell transplantation
Enzyme replacement therapy
Inborn errors of metabolism
Online Access:https://apcz.umk.pl/JEHS/article/view/57726
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