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Clinical Trials for Gangliosidosis, GM1

• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
• GM1 Prophylaxis for WBRT Related Cognitive Dysfunction
• A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type II GM1 Gangliosidosis
• Synergistic Enteral Regimen for Treatment of the Gangliosidoses
• HSCT for High Risk Inherited Inborn Errors
• Stem Cell Transplant for Inborn Errors of Metabolism