Natural History of Adult Patients with GM2 Gangliosidosis.

Marion MasingueLouis DufourTimothée LengletLisa SaleilleCyril GoizetXavier AyrignacFabienne Ory-MagneMagali BarthFoudil LamariDaniele MandiaCatherine CaillaudYann Nadjar

Published in: Annals of neurology (2020)

Improved knowledge of GM2 gangliosidosis in adults will help clinicians achieve correct diagnoses and better inform patients on the evolution and prognosis. It may also contribute to defining proper outcome measures when testing emerging therapies. ANN NEUROL 2020;87:609-617.

Keyphrases

end stage renal disease
newly diagnosed
ejection fraction
healthcare
chronic kidney disease
peritoneal dialysis
prognostic factors
palliative care