Article

Long-term Natural History and Elosulfase Alfa Treatment for Mucopolysaccharidosis Type IVA: A Single-Center Study in the Czech Republic

2026-03-26

Abstract excerpt

<title>Abstract</title> <p> <bold>Background:</bold> Mucopolysaccharidosis type IVA (MPS IVA, Morquio A syndrome) is a rare lysosomal storage disease primarily characterized by severe skeletal dysplasia. Clinical and laboratory data, including treatment responses, are reported for a cohort of Czech patients with MPS IVA. <bold>Results:</bold> Nineteen patients with MPS IVA from 17 families (13M/6F) were inclu...

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Literature Corpus work: c58bd765-76e2-5dea-955b-5b800867a0b2
DOI: 10.21203/rs.3.rs-9023839/v1

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