Overview

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Status:
Terminated
Trial end date:
2020-10-01
Target enrollment:
Participant gender:
Summary
This is a five-year extension study of the pilot study, "Intrathecal Enzyme Replacement for Cognitive Decline in MPS I". Participants must have completed the pilot study to participate in this study.
Phase:
N/A
Details
Lead Sponsor:
agnes chen
Collaborators:
Children's Hospital & Research Center Oakland
National Institute of Neurological Disorders and Stroke (NINDS)
Rare Diseases Clinical Research Network
The Ryan Foundation
UCSF Benioff Children's Hospital Oakland
University of California, Los Angeles
University of Minnesota
University of Minnesota - Clinical and Translational Science Institute