Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Status:
Terminated
Trial end date:
2011-10-01
Target enrollment:
Participant gender:
Summary
The investigators are studying the use of enzyme replacement therapy into the spinal fluid
for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of
mucopolysaccharidosis I (MPS I). Funding source -- FDA OOPD
Phase:
Phase 1
Details
Lead Sponsor:
Patricia I. Dickson, M.D.
Collaborators:
FDA Office of Orphan Products Development The Ryan Foundation University of California, Los Angeles