A Natural History Study of Canavan Disease
GTA-Glyceryltriacetate for Canavan Disease
Lithium and Acetate for Canavan Disease
Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
|5||Active, not recruiting||
Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
|7||Active, not recruiting||
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
|8||Active, not recruiting||
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders (UCDs)
† Study has passed its completion date and status has not been verified in more than two years.