The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 in patients who are undergoing standard …

AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more

~Estimated

~$10 – $7,500

Joanne Kurtzberg, MDNCT02254863

Longitudinal Study of Urea Cycle Disorders

Urea cycle disorders (UCD) are a group of rare inherited metabolism disorders. Infants and children with UCD commonly experience episodes of vomiting, lethargy…

Brain Diseases, Metabolic, InbornAmino Acid Metabolism, Inborn ErrorsUrea Cycle Disorders

~Estimated

~$10 – $1,125

Andrea GropmanNCT00237315