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

por Shapiro, Elsa G., Whitley, Chester B., Eisengart, Julie B.
Publicado 2018

The impact of COVID-19 on Brazilian children with MPS: Advocate group perspective

por Oliveira, Maria Carolina F.A., Yund, Brianna D., Grant, Nathan, Eisengart, Julie B., Giugliani, Roberto
Publicado 2022

Long-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome

por Eisengart, Julie B., Jarnes, Jeanine, Ahmed, Alia, Nestrasil, Igor, Ziegler, Richard, Delaney, Kathleen, Shapiro, Elsa, Whitley, Chester
Publicado 2017

Hematopoietic cell transplantation for sialidosis type I

por Gupta, Ashish O., Patterson, Marc C., Wood, Tim, Eisengart, Julie B., Orchard, Paul J., Lund, Troy C.
Publicado 2021

Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology

por Hampe, Christiane S., Eisengart, Julie B., Lund, Troy C., Orchard, Paul J., Swietlicka, Monika, Wesley, Jacob, McIvor, R. Scott
Publicado 2020

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

por Ahmed, Alia, Ou, Li, Rudser, Kyle, Shapiro, Elsa, Eisengart, Julie B., King, Kelly, Chen, Agnes, Dickson, Patricia, Whitley, Chester B.
Publicado 2019

Hematopoietic stem cell transplant for Hurler syndrome: does using bone marrow or umbilical cord blood make a difference?

por Orchard, Paul J., Gupta, Ashish O., Eisengart, Julie B., Polgreen, Lynda E., Pollard, Laura M., Braunlin, Elizabeth, Pasquali, Marzia, Lund, Troy C.
Publicado 2022

Differential outcomes for frontal versus posterior demyelination in childhood cerebral adrenoleukodystrophy

por Gupta, Ashish O., Nascene, David R., Shanley, Ryan, Kenney‐Jung, Daniel L., Eisengart, Julie B., Lund, Troy C., Orchard, Paul J., Pierpont, Elizabeth I.
Publicado 2021

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

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

Biochemical and clinical response after umbilical cord blood transplant in a boy with early childhood‐onset beta‐mannosidosis

por Lund, Troy C., Miller, Weston P., Eisengart, Julie B., Simmons, Katrina, Pollard, Laura, Renaud, Deborah L., Wenger, David A., Patterson, Marc C., Orchard, Paul J
Publicado 2019

Timing is everything: Clinical courses of Hunter syndrome associated with age at initiation of therapy in a sibling pair

por Grant, Nathan, Sohn, Young Bae, Ellinwood, N. Matthew, Okenfuss, Ericka, Mendelsohn, Bryce A., Lynch, Leslie E., Braunlin, Elizabeth A., Harmatz, Paul R., Eisengart, Julie B.
Publicado 2022

Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement

por Hampe, Christiane S., Wesley, Jacob, Lund, Troy C., Orchard, Paul J., Polgreen, Lynda E., Eisengart, Julie B., McLoon, Linda K., Cureoglu, Sebahattin, Schachern, Patricia, McIvor, R. Scott
Publicado 2021

Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy

por Pierpont, Elizabeth I., Nascene, David R., Shanley, Ryan, Kenney-Jung, Daniel L., Ziegler, Richard S., Miller, Weston P., Gupta, Ashish O., Lund, Troy C., Orchard, Paul J., Eisengart, Julie B.
Publicado 2020

Issues of COVID-19-related distance learning for children with neuronopathic mucopolysaccharidoses

por Eisengart, Julie B., Esler, Amy N., Ellinwood, N. Mathew, Hudock, Rebekah L., King, Kelly E., Klein, Terri L., Lee, Chimei, Morton, Jonathan, Stephens, Kim, Ziegler, Richard, O'Neill, Cara
Publicado 2021

Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation

por Polgreen, Lynda E., Lund, Troy C., Braunlin, Elizabeth, Tolar, Jakub, Miller, Bradley S., Fung, Ellen, Whitley, Chester B., Eisengart, Julie B., Northrop, Elise, Rudser, Kyle, Miller, Weston P., Orchard, Paul J.
Publicado 2019

Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I

por Lund, Troy C., Doherty, Terence M., Eisengart, Julie B., Freese, Rebecca L., Rudser, Kyle D., Fung, Ellen B., Miller, Bradley S., White, Klane K., Orchard, Paul J., Whitley, Chester B., Polgreen, Lynda E.
Publicado 2020

Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

por Eisengart, Julie B., Pierpont, Elizabeth I., Kaizer, Alexander M., Rudser, Kyle D., King, Kelly E., Pasquali, Marzia, Polgreen, Lynda E., Dickson, Patricia I., Le, Steven Q., Miller, Weston P., Tolar, Jakub, Orchard, Paul J., Lund, Troy C.
Publicado 2019

Long-term outcomes of systemic therapies for Hurler syndrome: an international multi-center comparison

por Eisengart, Julie B., Rudser, Kyle D., Xue, Yong, Orchard, Paul, Miller, Weston, Lund, Troy, Van der Ploeg, Ans, Mercer, Jean, Jones, Simon, Mengel, Karl Eugen, Gökce, Seyfullah, Guffon, Nathalie, Giugliani, Roberto, Souza, Carolina F. M., Shapiro, Elsa G., Whitley, Chester B.
Publicado 2018