Single Institutional Experience with GM1 Gangliosidosis: Clinical and Laboratory Results of 14 Patients

Halil Tuna Akar Yilmaz Yildiz Gökhan Güvenkaya Kismet Çıkı Ayça Burcu Kahraman Izzet Erdal Turgay Coşkun Hatice Serap Sivri Ayşegül Tokatlı

Published in: Balkan medical journal (2022)

Median survival in patients with GM1 gangliosidosis is less than 24 months. Beta-galactosidase enzyme activity may be associated with clinical onset and time of diagnosis in these patients.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
peritoneal dialysis
prognostic factors
patient reported outcomes
patient reported