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Enzymatic replacement therapy for Hunter disease: Up to 9 years experience with 17 patients

Hunter disease is an X-linked lysosomal storage disorder characterized by progressive storage of glycosaminoglycans (GAGs) and multi-organ impairment. The central nervous system (CNS) is involved in at least 50% of cases. Since 2006, the enzymatic replacement therapy (ERT) is available but with no e...

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Detalles Bibliográficos
Autores principales:	Parini, Rossella, Rigoldi, Miriam, Tedesco, Lucia, Boffi, Lucia, Brambilla, Alessandra, Bertoletti, Sara, Boncimino, Agata, Del Longo, Alessandra, De Lorenzo, Paola, Gaini, Renato, Gallone, Denise, Gasperini, Serena, Giussani, Carlo, Grimaldi, Marco, Grioni, Daniele, Meregalli, Pamela, Messinesi, Grazia, Nichelli, Francesca, Romagnoli, Marco, Russo, Pierluigi, Sganzerla, Erik, Valsecchi, Grazia, Biondi, Andrea
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2015
Materias:	SI:Therapy
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4750582/ https://www.ncbi.nlm.nih.gov/pubmed/26937399 http://dx.doi.org/10.1016/j.ymgmr.2015.03.011

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