Phenotype prediction for mucopolysaccharidosis type I by in silico analysis

by Li Ou, Michael J. Przybilla, Chester B. Whitley
Published 2017-07-01

Chitotriosidase as a biomarker for gangliosidoses

by Sarah Kim, Chester B. Whitley, Jeanine R. Jarnes
Published 2021-12-01

Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome

by Elsa G. Shapiro, Chester B. Whitley, Julie B. Eisengart
Published 2018-05-01

Genotype-phenotype correlation of gangliosidosis mutations using in silico tools and homology modeling

by Li Ou, Sarah Kim, Chester B. Whitley, Jeanine R. Jarnes-Utz
Published 2019-09-01

Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I

by Li Ou, Michael J. Przybilla, Brenda L. Koniar, Chester B. Whitley
Published 2016-09-01

The juvenile gangliosidoses: A timeline of clinical change

by Kelly E. King, Sarah Kim, Chester B. Whitley, Jeanine R. Jarnes-Utz
Published 2020-12-01

Hypothyroidism in late-onset Pompe disease

by Joseph Schneider, Lynn A. Burmeister, Kyle Rudser, Chester B. Whitley, Jeanine Jarnes Utz
Published 2016-09-01

Association of somatic burden of disease with age and neuropsychological measures in attenuated mucopolysaccharidosis types I, II and VI

by Alia Ahmed, Elsa Shapiro, Kyle Rudser, Alicia Kunin-Batson, Kelly King, Chester B. Whitley
Published 2016-06-01

Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population

by Christine Haller, Wenjie Song, Tricia Cimms, Chao‐Yin Chen, Chester B. Whitley, Raymond Y. Wang, Mislen Bauer, Paul Harmatz
Published 2019-09-01

Pilot study of the safety and effect of adalimumab on pain, physical function, and musculoskeletal disease in mucopolysaccharidosis types I and II

by Lynda E. Polgreen, Alicia Kunin-Batson, Kyle Rudser, Richard K. Vehe, Jeanine J. Utz, Chester B. Whitley, Patricia Dickson
Published 2017-03-01

A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation

by Alia Ahmed, Li Ou, Kyle Rudser, Elsa Shapiro, Julie B. Eisengart, Kelly King, Agnes Chen, Patricia Dickson, Chester B. Whitley
Published 2019-09-01

Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I

by Daniel A. Wolf, Andrew W. Lenander, Zhenhong Nan, Lalitha R. Belur, Chester B. Whitley, Pankaj Gupta, Walter C. Low, R. Scott McIvor
Published 2011-07-01

A longitudinal study of emotional adjustment, quality of life and adaptive function in attenuated MPS II

by Elsa G. Shapiro, Kyle Rudser, Alia Ahmed, Robert D. Steiner, Kathleen A. Delaney, Brianna Yund, Kelly King, Alicia Kunin-Batson, Julie Eisengart, Chester B. Whitley
Published 2016-06-01

Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis

by Michael J. Przybilla, Christine Stewart, Timothy W. Carlson, Li Ou, Brenda L. Koniar, Rohini Sidhu, Pamela J. Kell, Xuntian Jiang, Jeanine R. Jarnes, M. Gerard O'Sullivan, Chester B. Whitley
Published 2021-06-01

Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I

by Troy C. Lund, Terence M. Doherty, Julie B. Eisengart, Rebecca L. Freese, Kyle D. Rudser, Ellen B. Fung, Bradley S. Miller, Klane K. White, Paul J. Orchard, Chester B. Whitley, Lynda E. Polgreen
Published 2021-03-01