Mucopolysaccharidosis type VI on enzyme replacement therapy since infancy: Six years follow-up of four children()

Clinical and biochemical improvements are reported on Mucopolysaccharidosis type VI (MPS VI) patients on Enzyme Replacement Therapy (ERT) with rhASB (galsulfase, Naglazyme®), and preclinical and clinical studies have shown clinical benefits of early initiation. We report four unrelated MPS VI childr...

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Detalles Bibliográficos
Autores principales: Horovitz, Dafne D.G., Acosta, Angelina Xavier, de Rosso Giuliani, Liane, Ribeiro, Erlane Marques
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5471404/
https://www.ncbi.nlm.nih.gov/pubmed/28649537
http://dx.doi.org/10.1016/j.ymgmr.2015.09.002

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