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

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…

LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more

~Estimated

~$10 – $750

Children's Hospital of PhiladelphiaNCT03047369