Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
Status:
Terminated
Trial end date:
2018-05-01
Target enrollment:
Participant gender:
Summary
This is a standard of care treatment guideline for patients with the diagnosis of
mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates
for first hematopoietic stem cell transplantation (HSCT) according to a University of
Minnesota myeloablative HSCT protocol.