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

Title

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

Authors

Keywords

mucopolysaccharidosis II, Hunter syndrome, pabinafusp alfa, blood brain barrier, iduronate-2-sulfatase, central nervous system, neurocognitive development, neurocognitive impairment, neurodegeneration, anti-human transferrin receptor antibody, heparan sulfate, enzyme-replacement therapy

Journal

MOLECULAR THERAPY

Volume -, Issue -, Pages -

Publisher

Elsevier BV

Online

2020-09-30

DOI

10.1016/j.ymthe.2020.09.039