Cardiac manifestations and effects of enzyme replacement therapy for over 10 years in adults with the attenuated form of mucopolysaccharidosis type I

Background: Mucopolysaccharidosis type I (MPS I) is a rare autosomal recessive disease caused by a deficiency of the lysosomal enzyme α-L-iduronidase. Cardiac manifestations such as valvular heart disease are associated with poor prognosis. There have been only a few reports on the effect of long-te...

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Bibliografiske detaljer

Principais autores:	Kenta Sugiura, Toru Kubo, Yuri Ochi, Yuichi Baba, Takayoshi Hirota, Naohito Yamasaki, Hiroaki Kitaoka
Format:	Artigo
Sprog:	Inglês
Udgivet:	Elsevier 2020-12-01
Serier:	Molecular Genetics and Metabolism Reports
Fag:	Mucopolysaccharidosis Scheie syndrome Enzyme replacement therapy Adults Valvular disease
Online adgang:	http://www.sciencedirect.com/science/article/pii/S2214426920301087
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