GM1 Gangliosidosis Type II: Results of a 10-Year Prospective Study
Precilla D’Souza, Cristan Farmer, Jean M. Johnston, Sangwoo T. Han, David R. Adams, Adam L. Hartman, Wadih M. Zein, Laryssa A. Huryn, Beth I. Solomon, Kelly A. King, Christopher Phillips Jordan, Jennifer G. Myles, Elena‐Raluca Nicoli, Caroline E Rothermel, Yoliann Mojica Algarin, , Cynthia J. Tifft
This study presents a 10-year prospective analysis of type II GM1 gangliosidosis, detailing disease progression and phenotypic features in 41 patients.
Genetics in Medicine · Apr 16, 2024Peer-reviewedGenetic & Genomic Medicine