2 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
Interventional
TRIAL ROLE: Co-Investigator
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A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease
The purpose of this study is to find out if an investigational new drug called ATB200 and AT2221 can help people with Pompe disease and ...Phase
Interventional
TRIAL ROLE: Sub Investigator
