Mucopolysaccharidosis Type II Observational

key information

study id #: NCT04591834

condition: Mucopolysaccharidosis II

status: Recruiting


This is an observational study planned to document prospectively disease manifestation and neurocognitive course in pediatric patients with a clinical presentation consistent with neuronopathic (“severe”) MPS II undergoing current standard of care and/or intrathecal Elaprase® for their condition. Some patients may be offered the opportunity to screen for a gene therapy study conducted by the same sponsor.

intervention: Observational


last updated: February 26, 2022