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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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Genet Metab Rep
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Elsevier 2015
Pynciau:
SI:Therapy
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4750582/
https://ncbi.nlm.nih.gov/pubmed/26937399
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2015.03.011
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