Mucopolysaccharidosis Type I (Hurler – Scheie Syndrome): Case Report

Background. Mucopolysaccharidosis type I (MPS I) is an inherited disease caused by pathogenic variants in the IDUA gene, which encodes the lysosomal enzyme alpha-L-iduronidase. This clinical case demonstrates the importance of diagnosing rare diseases when patients have phenotypic features indicatin...

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Bibliographic Details
Main Authors: Nato D. Vashakmadze, Olga I. Gumenuyk, Yuri V. Chernenkov, Ekaterina Yu. Zacharova, Igor A. Glushakov
Format: Article
Language:English
Published: "Paediatrician" Publishers LLC 2024-09-01
Series:Вопросы современной педиатрии
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Online Access:https://vsp.spr-journal.ru/jour/article/view/3568
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