K. Vondrak

Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

P. Fitzpatrick, S. Bunting, A. Bagri, 2014, Proceedings of the National Academy of Sciences.

A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I

M. Iacovino, J. Cooper, M. Sands, 2017, Molecular therapy. Methods & clinical development.

Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I

James M. Provenzale, Igor Nestrasil, David Elashoff, 2015, Experimental Neurology.

Combined adenine phosphoribosyltransferase and N-acetylgalactosamine-6-sulfate sulfatase deficiency.

H. Simmonds, J. Tischfield, J. Zeman, 1999, Molecular genetics and metabolism.