Compassionate Use: A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
The approved drug, aldurazyme, will be injected into the spinal canal via Lumbar Puncture. The patient will be treated once every 30 days for 3 months and then once every 3 months for 4 years. The patient will be monitored for side effects and kept in 23 hour observation post procedure. The uploaded protocol (active at uCLa) will be followed for procedure and safety purposes.
*presence of MPS I disease
*minimum 6 months consecutive IV ERT with Aldurazyme
*18 years of age
*acquired cognitive deficits