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Profile of idursulfase for the treatment of Hunter syndrome

Authors Sestito S, Ceravolo F, Grisolia M, Pascale E, Pensabene L, Concolino D

Received 20 March 2015

Accepted for publication 5 May 2015

Published 9 July 2015 Volume 2015:5 Pages 79—90


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 4

Editor who approved publication: Professor Mingzhao Xing

Simona Sestito, Ferdinando Ceravolo, Michele Grisolia, Elisa Pascale, Licia Pensabene, Daniela Concolino

 Department of Pediatrics, University Magna Graecia of Catanzaro, Catanzaro, Italy

Abstract: Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X-linked lysosomal storage disorder caused by deficiency of the enzyme iduronate-2-sulfatase (IDS). Enzyme replacement therapy (ERT) with recombinant human IDS, available since 2005, is currently the most appropriate treatment for this progressive, multisystemic, chronic, and life-threatening disease. Efficacy and safety of therapy with idursulfase have been assessed in several clinical trials, and confirmed in many clinical reports. Long-term follow-up of patients receiving ERT has demonstrated the importance of an early onset of treatment with idursulfase, before irreversible pathological changes occur. Intravenously administered idursulfase is not able to cross the blood–brain barrier, so neurological signs and symptoms cannot benefit from ERT, still remaining a major challenge in the treatment of MPS II.

Keywords: MPS II, glycosaminoglycans, enzyme replacement therapy, ERT

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