1 clinical trials found.
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Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational multicenter open-label rater-blinded Phase II study.
The purpose of this study is to investigate if the study drug N-Acetyl-L-Leucine, called IB1001, can help improve the symptoms of GM2 Gangliosidosis after 6-weeks ...Phase
TRIAL ROLE: Co-Investigator
