Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients von Giugliani, R., Harmatz, P., Jones, S.A., Mendelsohn, N.J., Vellodi, A., Qiu, Y., Hendriksz, C.J., Vijayaraghavan, S., Whiteman, D.A.H., Pano, A.

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PSY33 Item Reduction and Assessment of Measurement Properties of the Hunter Syndrome – Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) von Wiklund, I, Raluy, M, Chen, W.H, Muenzer, J, Pelletier, N, Fang, J, Whiteman, D.A.H

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PIH40 Designing a Disease-Specific Patient Reported Outcome (PRO) for a Rare Disease: Assessing the Reliability, Validity, and Responsiveness of the Hunter Syndrome - Functional Ou... von Wiklund, I, Raluy, M, Jangelind, Y, Whiteman, D.A.H, Conway, A.M, Pang, F, Stull, D.E, Chen, W.H

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The relationship between anti-idursulfase antibody status and safety and efficacy outcomes in attenuated mucopolysaccharidosis II patients aged 5years and older treated with intrav... von Barbier, A.J., Bielefeld, B., Whiteman, D.A.H., Natarajan, M., Pano, A., Amato, D.A.

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Molecular characterization of cytogenetic alterations associated with the Beckwith — Wiedemann syndrome (BWS) phenotype refines the localization and suggests the gene for BWS is im... von Weksberg, R., Teshima, I., Williams, B.R.G., Greenberg, C.R., Pueschel, S.M., Chernos, J.E., Fowlow, S.B., Hoyme, E., Anderson, I.J., Whiteman, D.A.H., Fisher, N., Squire, J.

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