Genetic & Genomic MedicineGenetics in MedicinePeer-reviewed
GM1 Gangliosidosis Type II: Results of a 10-Year Prospective Study
Precilla D’Souza, Cristan Farmer, Jean M. Johnston…·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.
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