Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil

Novel Enzyme Replacement Therapies for Neuropathic Mucopolysaccharidoses

(2020) Yuji Sato et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II

(2020) Torayuki Okuyama et al.   MOLECULAR THERAPY

Natural history of cognitive development in neuronopathic mucopolysaccharidosis type II (Hunter syndrome): Contribution of genotype to cognitive developmental course

(2020) Joo-Hyun Seo et al.   Molecular Genetics and Metabolism Reports

Transferrin and transferrin receptors update

(2018) Hiroshi Kawabata   FREE RADICAL BIOLOGY AND MEDICINE

A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II

(2018) Hiroyuki Sonoda et al.   MOLECULAR THERAPY

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

(2018) Roberto Giugliani et al.   Orphanet Journal of Rare Diseases

Blood–brain barrier opening in Alzheimer’s disease using MR-guided focused ultrasound

(2018) Nir Lipsman et al.   Nature Communications

Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC/MS/MS

(2018) Noboru Tanaka et al.   MOLECULAR GENETICS AND METABOLISM

Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial

(2018) Torayuki Okuyama et al.   MOLECULAR THERAPY

Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses

(2017) Elsa G. Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

Hyperthermic Laser Ablation of Recurrent Glioblastoma Leads to Temporary Disruption of the Peritumoral Blood Brain Barrier

(2016) Eric C. Leuthardt et al.   PLoS One

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

(2015) Joseph Muenzer et al.   GENETICS IN MEDICINE

Insulin receptor antibody-iduronate 2-sulfatase fusion protein: Pharmacokinetics, anti-drug antibody, and safety pharmacology in Rhesus monkeys

(2014) Ruben J. Boado et al.   BIOTECHNOLOGY AND BIOENGINEERING

Increased Brain Penetration and Potency of a Therapeutic Antibody Using a Monovalent Molecular Shuttle

(2014) Jens Niewoehner et al.   NEURON

Therapeutic bispecific antibodies cross the blood-brain barrier in nonhuman primates

(2014) Y. J. Yu et al.   Science Translational Medicine

Addressing Safety Liabilities of TfR Bispecific Antibodies That Cross the Blood-Brain Barrier

(2013) J. A. Couch et al.   Science Translational Medicine

Therapeutic Potential of Intracerebroventricular Replacement of Modified Human β-Hexosaminidase B for GM2 Gangliosidosis

(2011) Kazuhiko Matsuoka et al.   MOLECULAR THERAPY

Boosting Brain Uptake of a Therapeutic Antibody by Reducing Its Affinity for a Transcytosis Target

(2011) Y. J. Yu et al.   Science Translational Medicine

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

(2010) Joseph Muenzer et al.   GENETICS IN MEDICINE

Japan Elaprase® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)

(2009) Torayuki Okuyama et al.   MOLECULAR GENETICS AND METABOLISM