Litcius/Paper detail

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

2024Genetics in Medicine18 citationsDOIOpen Access PDF

Topics & Concepts

MedicinePediatricsDiseaseHepatosplenomegalyMucopolysaccharidosis type IJuvenileProspective cohort studyGangliosidosisEnzyme replacement therapyInternal medicineBiologyGeneticsLysosomal Storage Disorders ResearchGlycosylation and Glycoproteins ResearchCellular transport and secretion