Gangliosidoses - 9 Studies Found
Completed |
: Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis : Gangliosidoses GM2 : 2007-01-04 : Drug: miglustat Target dose of 320 mg/m^2/day (divided in 3 doses) will be based on the Body Surface Are |
Completed |
: Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff 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 |
: A Natural History of Late Onset Tay-Sachs Disease : GM2 Gangliosidosis : 2016-07-19 |
Withdrawn |
: A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis :
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Completed |
: Gene Therapy for Tay-Sachs Disease :
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Completed |
: Diagnostic and Screening Study of Genetic Disorders :
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Active, not recruiting |
: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders :
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Withdrawn |
: Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases :
: 2009-10-20 : Biological: ALD-601 ALD-601 is manufactured by Aldagen from the 20% compartment of the selected umbilica |