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| Found 7 studies with search of: | "Sandhoff Disease" |
| Rank | Status | Study | ||||
|---|---|---|---|---|---|---|
| 1 | Suspended |
A Phase I Study of Pyrimethamine in Patients With GM-2 Gangliosidosis
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| 2 | Completed |
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
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| 3 | Recruiting |
HSCT for High Risk Inherited Inborn Errors
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| 4 | Active, not recruiting |
Stem Cell Transplant for Inborn Errors of Metabolism
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| 5 | Recruiting |
Nervous System Degeneration in Glycosphingolipid Storage Disorders
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| 6 | Suspended |
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
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| 7 | Active, not recruiting |
Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
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