Key result
Enzyme replacement therapy fails to improve mitral valve disease severity in mucopolysaccharidosis type VI.
Why the study?
Cardiac involvement in patients with mucopolysaccharidosis type VI, including valve abnormalities, requires evaluation to guide monitoring and treatment.
Observational (n=13)
No
p-value: p=>0.05
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Emine Azak (2019) conducted an observational in Mucopolysaccharidosis type VI (n=13). Enzyme replacement therapy (galsulfase) vs. No enzyme replacement therapy was evaluated on Differences in severity of valve regurgitation (p=>0.05). Mitral valve deformation is the most frequent cardiac finding in children with mucopolysaccharidosis type VI, and enzyme replacement therapy does not significantly affect valve involvement severity.
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