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Clinical Trials for Gangliosidosis, GM1

  • 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
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