Analysis of Caregiver Perspectives on Patients With Mucopolysaccharidosis II Treated With Pabinafusp Alfa: Results of Qualitative Interviews in Japan [PDF]
Orphanet Journal of Rare DiseasesBackground Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a rare X-linked metabolic disorder predominantly affecting males. Pabinafusp alfa, an iduronate-2-sulfatase enzyme designed to cross the blood-brain barrier, was approved in Japan ...
Yuji Sato +2 more
exaly +6 more sources
Enzyme Replacement Therapy With Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data [PDF]
International Journal of Molecular Sciences, 2021 Enzyme replacement therapy (ERT) improves the somatic manifestations in mucopolysaccharidoses (MPS))．However, because intravenously administered enzymes cannot cross the blood brain barrier (BBB), ERT is ineffective against the progressive neurodegeneration and resultant severe central nervous system (CNS) symptoms observed in patients with ...
Roberto Giugliani +2 more
exaly +10 more sources
Transferrin Receptor-Targeting Property of Pabinafusp Alfa Facilitates Its Uptake by Various Types of Human Brain-Derived Cells in Vitro [PDF]
Frontiers in Drug Delivery, 2023 Pabinafusp alfa, which is an anti-mucopolysaccharidosis II drug, consists of iduronate-2-sulfatase (IDS) genetically fused with an anti-transferrin receptor (TfR) antibody.
Tomomi Furihata +2 more
exaly +6 more sources
Iduronate-2-Sulfatase Fused With Anti-hTfR Antibody, Pabinafusp Alfa, for MPS-II: A Phase 2 Trial in Brazil [PDF]
Molecular Therapy, 2021 In Hunter syndrome (mucopolysaccharidosis II [MPS-II]), systemic accumulation of glycosaminoglycans (GAGs) due to a deficiency of iduronate-2-sulfatase (IDS), caused by mutations in the IDS gene, leads to multiple somatic manifestations and in patients with the severe (neuronopathic) phenotype, also to central nervous system (CNS) involvement.
Ana Maria Guerra Martins +2 more
exaly +8 more sources
Nonclinical Safety Evaluation of Pabinafusp Alfa, an Anti-Human Transferrin Receptor Antibody and Iduronate-2-Sulfatase Fusion Protein, for the Treatment of Neuronopathic Mucopolysaccharidosis Type II [PDF]
Molecular Genetics and Metabolism Reports, 2021 Pabinafusp alfa is a fusion protein comprising a humanized anti-human transferrin receptor (TfR) antibody and human iduronate-2-sulfatase. It was developed as a novel modality to target central nervous system-related symptoms observed in patients with ...
Kohtaro Minami +2 more
exaly +6 more sources
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused With Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II [PDF]
Molecular Therapy, 2021 Pabinafusp alfa (JR-141) is a novel enzyme drug that crosses the blood-brain barrier by transcytosis via transferrin receptors. In order to establish its efficacy and safety, a multicenter, single-arm, open-label phase 2/3 clinical trial was conducted in 28 Japanese patients with mucopolysaccharidosis II (MPS-II, Hunter syndrome) by intravenous ...
Yuji Sato, Torayuki Okuyama, Norio Sakai
exaly +6 more sources
Divergent developmental trajectories in two siblings with neuropathic mucopolysaccharidosis type II (Hunter syndrome) receiving conventional and novel enzyme replacement therapies: A case report [PDF]
JIMD Reports, 2021 Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X‐linked recessive lysosomal storage disease caused by a mutation in the IDS gene and characterized by systemic accumulations of glycosaminoglycans.
Kazuyoshi Tomita +8 more
doaj +3 more sources
Infusion Rate Adjustment in Enzyme Replacement Therapy With Pabinafusp Alfa for Mucopolysaccharidosis II [PDF]
British Journal of Clinical PharmacologyAbstract Aims Enzyme replacement therapy (ERT) for mucopolysaccharidosis II (MPS II) requires long‐term, weekly intravenous infusions often lasting over 3 h each time, which can burden paediatric patients and caregivers and negatively affect their quality of life and treatment compliance.
Yuji Sato +2 more
exaly +3 more sources
Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice [PDF]
Molecular Therapy: Methods & Clinical Development, 2022 Deposition of heparan sulfate (HS) in the brain of patients with mucopolysaccharidosis II (MPS II) is believed to be the leading cause of neurodegeneration, resulting in several neurological signs and symptoms, including neurocognitive impairment.
Hideto Morimoto +6 more
doaj +2 more sources
Heparan sulfate in cerebrospinal fluid as a biomarker to assess disease severity and for treatment monitoring in patients with Mucopolysaccharidosis Type II: a position statement [PDF]
Orphanet Journal of Rare DiseasesPatients with mucopolysaccharidosis type II (MPS II) can present with a severe neuronopathic phenotype or an attenuated non-neuronopathic phenotype.
Roberto Giugliani +7 more
doaj +2 more sources