Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial

Plasma Pharmacokinetics of Valanafusp Alpha, a Human Insulin Receptor Antibody-Iduronidase Fusion Protein, in Patients with Mucopolysaccharidosis Type I

(2018) William M. Pardridge et al.   BIODRUGS

Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome

(2018) Elsa G. Shapiro et al.   Orphanet Journal of Rare Diseases

Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

(2017) Marc Tardieu et al.   LANCET NEUROLOGY

Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure

(2017) Johanna H. van der Lee et al.   MOLECULAR GENETICS AND METABOLISM

Reduction in Brain Heparan Sulfate with Systemic Administration of an IgG Trojan Horse–Sulfamidase Fusion Protein in the Mucopolysaccharidosis Type IIIA Mouse

(2017) Ruben J. Boado et al.   MOLECULAR PHARMACEUTICS

Brain and Organ Uptake in the Rhesus Monkey in Vivo of Recombinant Iduronidase Compared to an Insulin Receptor Antibody–Iduronidase Fusion Protein

(2017) Ruben J. Boado et al.   MOLECULAR PHARMACEUTICS

Effect of anti-laronidase antibodies on efficacy and safety of laronidase enzyme replacement therapy for MPS I: A comprehensive meta-analysis of pooled data from multiple studies

(2016) Yong Xue et al.   MOLECULAR GENETICS AND METABOLISM

A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA

(2016) Simon A. Jones et al.   MOLECULAR GENETICS AND METABOLISM

Very High Plasma Concentrations of a Monoclonal Antibody against the Human Insulin Receptor Are Produced by Subcutaneous Injection in the Rhesus Monkey

(2016) Ruben J. Boado et al.   MOLECULAR PHARMACEUTICS

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

(2015) M. Aldenhoven et al.   BLOOD

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

(2015) Joseph Muenzer et al.   GENETICS IN MEDICINE

Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment

(2015) Elsa G. Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

IgG-Enzyme Fusion Protein: Pharmacokinetics and Anti-Drug Antibody Response in Rhesus Monkeys

(2012) Ruben J. Boado et al.   BIOCONJUGATE CHEMISTRY

Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats

(2012) Dyane Auclair et al.   PEDIATRIC RESEARCH

Analysis of Glycosaminoglycans in Cerebrospinal Fluid from Patients with Mucopolysaccharidoses by Isotope-Dilution Ultra-Performance Liquid Chromatography-Tandem Mass Spectrometry

(2011) H. Zhang et al.   CLINICAL CHEMISTRY

Reversal of Lysosomal Storage in Brain of Adult MPS-I Mice with Intravenous Trojan Horse-Iduronidase Fusion Protein

(2011) Ruben J. Boado et al.   MOLECULAR PHARMACEUTICS

Recognizing Hypoglycemia in Children Through Automated Adverse-Event Detection

(2011) M. J. Dickerman et al.   PEDIATRICS

Correlation of Automated Volumetric Analysis of Brain MR Imaging with Cognitive Impairment in a Natural History Study of Mucopolysaccharidosis II

(2010) Z. Fan et al.   AMERICAN JOURNAL OF NEURORADIOLOGY

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

(2010) Joseph Muenzer et al.   GENETICS IN MEDICINE

AGT-181: Expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys

(2009) Ruben J. Boado et al.   JOURNAL OF BIOTECHNOLOGY

A dose-optimization trial of laronidase (Aldurazyme®) in patients with mucopolysaccharidosis I

(2008) Roberto Giugliani et al.   MOLECULAR GENETICS AND METABOLISM