A Study To Evaluate The Effects Of N-Acetyl-L-Leucine For GM2 Gangliosdisosis (Tay-Sachs And Sandhoff Disease)
Rochester, MN
The purpose of this study is to assess the safety and effectiveness of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).