P039: Mucopolysaccharidoses: A case series of five pediatric cardiology patients in Marrakech, Morocco

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Authors: Kristina M. Woodis, Lucas Miecho Heilbroner, Drissi Boumzebra, Nadia El Idrissi Slitine, Christina Yang, Xinyang Liu, Faisal Al Munajjed, Ichrak Rajil, Craig Sable, Marius Linguraru, Christina Lee Grant
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Abstract

Mucopolysaccharidoses (MPS) are lysosomal storage disorders caused by deficiencies in enzymes that lead to the accumulation of glycosaminoglycans (GAGs) in different tissues, leading to variable presentation including skeletal dysostosis, dysmorphisms, cardiac disease, organomegaly, and neurodegeneration. Specific presentation depends on which enzyme is deficient with significant clinical overlap between the forms. Globally, patients with MPS have been reported in 33 countries in North America, South America, Africa, Europe, Asia, and Australia with a geographical and ethnic variance in incidence.

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Biomedical Research and Pathophysiology

Publication details

Journal / source
Genetics in Medicine Open
Volume
4
Pages
103530–103530
Publication date
1 January 2026
Language
EN