Intrathecal Enzyme Replacement for Hurler Syndrome
Status:
Completed
Trial end date:
2018-11-18
Target enrollment:
Participant gender:
Summary
This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered
into the spinal fluid of patients with Hurler syndrome at intervals before and after bone
marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen
in Hurler patients undergoing transplantation.