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Mucopolysaccharidosis Type II Observational

REGENXBIO Inc. · 2022
ClinicalTrials.gov · Datasets · License: Public Domain · 2022
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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.
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