Article
The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus.
European journal of pediatrics - 1 Jan 2012
Muenzer Joseph, Bodamer Olaf, Burton Barbara, Clarke Lorne, Frenking Gudrun Schulze, Giugliani Roberto, Jones Simon, Rojas Maria Verónica Muñoz, Scarpa Maurizio, Beck Michael, Harmatz Paul
Abstract excerpt
UNLABELLED: Intravenous enzyme replacement therapy (ERT) with idursulfase for Hunter syndrome has not been demonstrated to and is not predicted to cross the blood-brain barrier. Nearly all published experience with ERT with idursulfase has therefore been in patients without cognitive impairment (attenuated phenotype). Little formal guidance is available on the issues surrounding ERT in cognitively impaired...
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