Kazunori Tanizawa

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

Torayuki Okuyama, Yoshikatsu Eto, Norio Sakai et al. · Molecular Therapy · 2020 · 173 citations · Full text

Medicine, Neurology, Pharmacology +17

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

Roberto Giugliani, Ana Maria Martins, Sairei So et al. · Molecular Therapy · 2021 · 61 citations · Full text

Life Sciences, Neurology, Neuroscience +11

Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data

Roberto Giugliani, Ana Maria Martins, Torayuki Okuyama et al. · International Journal of Molecular Sciences · 2021 · 44 citations · Full text

Medicine, Neurology, Pharmacology +17

Divergent developmental trajectories in two siblings with neuropathic mucopolysaccharidosis type <scp>II</scp> (Hunter syndrome) receiving conventional and novel enzyme replacement therapies: A case report

Kazuyoshi Tomita, Shungo Okamoto, Toshiyuki Seto et al. · JIMD Reports · 2021 · 18 citations · Full text

Life Sciences, Genetics, Neurology +17

α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial

Paul Harmatz, Roberto Giugliani, Ana Maria Martins et al. · Molecular Therapy · 2024 · 14 citations · Full text

Medicine, Neurology, Neuroscience +15