Intrathecal Enzyme Replacement for Hurler Syndrome

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.

Timeline

Start

2008-01-02

Primary completion

2017-02-18

Completion

2018-11-18

Drugs

Indications