Sandhoff Disease - 14 Studies Found
Completed |
: Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) :
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Completed |
: Gene Therapy for Tay-Sachs Disease :
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Completed |
: Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses :
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Recruiting |
: Synergistic Enteral Regimen for Treatment of the Gangliosidoses :
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Recruiting |
: A Natural History Study of the Gangliosidoses :
: 2014-10-23 |
Completed |
: HSCT for High Risk Inherited Inborn Errors :
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Recruiting |
: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells :
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Active, not recruiting |
: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders :
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