Non-cardiac Manifestations in Adult Patients With Mucopolysaccharidosis.
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ABSTRACT:
SUBMITTER: Stepien KM
PROVIDER: S-EPMC8935042 | biostudies-literature | 2022
REPOSITORIES: biostudies-literature
Publications
Non-cardiac Manifestations in Adult Patients With Mucopolysaccharidosis.
Stepien Karolina M KM Bentley Andrew A Chen Cliff C Dhemech M Wahab MW Gee Edward E Orton Peter P Pringle Catherine C Rajan Jonathan J Saxena Ankur A Tol Govind G Gadepalli Chaitanya C
Frontiers in cardiovascular medicine 20220307
Mucopolysaccharidoses (MPS) are a heterogeneous group of disorders that results in the absence or deficiency of lysosomal enzymes, leading to an inappropriate storage of glycosaminoglycans (GAGs) in various tissues of the body such as bones, cartilage, heart valves, arteries, upper airways, cornea, teeth, liver and nervous system. Clinical manifestations can become progressively exacerbated with age and affect their quality of life. Developments in advanced supportive treatment options such as e ...[more]
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