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Gangliosidoses - 36 Studies Found

Status Study
Completed Study Name: Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Condition: Gangliosidoses GM2
Date: 2007-01-04
Interventions: Drug: miglustat Target dose of 320 mg/m^2/day (divided in 3 doses) will be based on the Body Surface Are
Completed Study Name: Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Condition:
  • Gangliosidoses, GM2
  • Sandhoff Disease
  • Drug: Pyrimethamine Pyrimetham
Recruiting Study Name: Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Condition:
  • GM1 Gangliosidoses
  • GM2 Gangliosidoses Date: 2013-12-17
    Interventions:
    • Drug: miglustat The Syner-G th
Completed Study Name: Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
Condition:
  • GM2 Gangliosidoses
  • Tay-Sachs

  • Date: 2008-05-02
    Interventions: Drug: Zavesca (Miglustat)
Recruiting Study Name: A Natural History Study of the Gangliosidoses
Condition:
  • Tay-Sachs Disease
  • Sandhoff Disease
  • Late Onset Tay-Sachs Di
    Date: 2008-04-25
Recruiting Study Name: A Natural History of Late Onset Tay-Sachs Disease
Condition: GM2 Gangliosidosis
Date: 2016-07-19
Recruiting Study Name: Biomarker for GM1/GM2 - Gangliosidoses
Condition:
  • Gangliosidosis
  • GM1-Gangliosidosis<
    Date: 2014-10-23
Recruiting Study Name: Nervous System Degeneration in Glycosphingolipid Storage Disorders
Condition:
  • Gangliosidoses
  • Gaucher Disease

Date: 2002-01-27
Withdrawn Study Name: A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis
Condition:
  • G(M2) Ganglioside
  • Tay-Sachs Disease Ganglioside
  • Sandhoff D
    Date: 2008-05-15
    Interventions: Drug: Pyrimethamine Pyrimethamine will be given PO once daily for 8 consecutive weeks.
Completed Study Name: HSCT for High Risk Inherited Inborn Errors
Condition:
  • Adrenoleukodystrophy
  • Metachromatic Leukodystrophy
  • Globoid
    Date: 2006-09-29
    Interventions:
    • Drug: Clofarabine days -7 thro
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