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
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Title
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
Authors
Keywords
Mucopolysaccharidosis Type I, Iduronidase, Blood-brain barrier, Insulin receptor, Open label clinical trial, Safety, Efficacy
Journal
Orphanet Journal of Rare Diseases
Volume 13, Issue 1, Pages -
Publisher
Springer Nature
Online
2018-07-05
DOI
10.1186/s13023-018-0849-8
References
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Note: Only part of the references are listed.- 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
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