A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5years and older treated with intravenous idursulfase

(2013) A.J. Barbier et al.   MOLECULAR GENETICS AND METABOLISM

Physiology of the Intrathecal Bolus: The Leptomeningeal Route for Macromolecule and Particle Delivery to CNS

(2013) Mikhail I. Papisov et al.   MOLECULAR PHARMACEUTICS

CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder

(2012) Pericles Calias et al.   PLoS One

The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus

(2011) Joseph Muenzer et al.   EUROPEAN JOURNAL OF PEDIATRICS

Early Clinical Markers of Central Nervous System Involvement in Mucopolysaccharidosis Type II

(2011) Joshua Holt et al.   JOURNAL OF PEDIATRICS

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

(2010) Joseph Muenzer et al.   GENETICS IN MEDICINE

Multidisciplinary Management of Hunter Syndrome

(2009) J. Muenzer et al.   PEDIATRICS

Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome)

(2008) R. Martin et al.   PEDIATRICS