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

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