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Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI: 10-Year follow up

Early initiation of enzyme replacement therapy (ERT) has demonstrated clinical benefit in patients with mucopolysaccharidosis type VI (MPS VI), a progressive, multisystem autosomal recessive lysosomal disorder caused by N-acetylgalactosamine-4-sulphatase (ASB) deficiency and the consequent accumulat...

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Detalles Bibliográficos
Autores principales:	Furujo, Mahoko, Kosuga, Motomichi, Okuyama, Torayuki
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2017
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5622997/ https://www.ncbi.nlm.nih.gov/pubmed/28983456 http://dx.doi.org/10.1016/j.ymgmr.2017.08.007

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