Sandhoff disease

Search Trials
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
Status:
Completed
Last Changed:
May 6, 2008
First Changed:
May 6, 2008
Disease(s):
GM2 Gangliosidoses
Intervention(s):
Zavesca (Miglustat)
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Status:
Active, not recruiting
Last Changed:
Sep 12, 2019
First Changed:
Jun 13, 2011
Disease(s):
Hurler Syndrome (MPS I)
Intervention(s):
hematopoietic stem cell infusion
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Status:
Terminated
Last Changed:
Jul 8, 2014
First Changed:
Apr 8, 2008
Disease(s):
Inherited Metabolic Diseases
Intervention(s):
ALD-101
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Status:
Recruiting
Last Changed:
May 29, 2019
First Changed:
Jan 8, 2014
Disease(s):
GM1 Gangliosidoses
Intervention(s):
miglustatKetogenic Diet
A Natural History Study of the Gangliosidoses
Status:
Recruiting
Last Changed:
May 29, 2019
First Changed:
Apr 29, 2008
Disease(s):
Tay-Sachs Disease

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