BENEFICIAL EFFECTS OF ACETYL-DL-LEUCINE (ADLL) IN A MOUSE MODEL OF SANDHOFF DISEASE

In Vivo studies with Acetyl-leucine significantly slowed disease progression and improved motor function in the GM2 gangliosidoses Sandhoff mouse model (Hexb-/-), supportive of the symptomatic and disease-modifying potential of treatment for patients with GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease).

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