GM1 gangliosidosis type II: Results of a 10-year prospective study
Precilla D’Souza, Cristan Farmer, Jean M. Johnston, Sangwoo T. Han, David Adams, Adam L. Hartman, Wadih M. Zein, Laryssa A. Huryn, Beth Solomon, Kelly King, Christopher P. Jordan, Jennifer Myles, Elena‐Raluca Nicoli, Caroline E Rothermel, Yoliann Mojica Algarin, Reyna Huang, Rachel Quimby, Mosufa Zainab, S. Bowden, Anna Crowell, Ashura Buckley, Carmen C. Brewer, Debra S. Regier, Brian P. Brooks, Maria T. Acosta, Eva H. Baker, Gilbert Vézina, Audrey Thurm, Cynthia J. Tifft
Topics & Concepts
MedicinePediatricsDiseaseHepatosplenomegalyMucopolysaccharidosis type IJuvenileProspective cohort studyGangliosidosisEnzyme replacement therapyInternal medicineBiologyGeneticsLysosomal Storage Disorders ResearchGlycosylation and Glycoproteins ResearchCellular transport and secretion