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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| Main Authors: | , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Kazimierz Wielki University
2025-02-01
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| Series: | Journal of Education, Health and Sport |
| Subjects: | |
| Online Access: | https://apcz.umk.pl/JEHS/article/view/57726 |
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