Mucopolysaccharidosis Type II Observational
study id #: NCT04591834
condition: Mucopolysaccharidosis II
status: Recruiting
purpose: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
results: https://clinicaltrials.gov/ct2/show/results/NCT04591834
last updated: February 26, 2022
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Walla Al-Hertani, MD, MS, FRCPC, FCCMG, FACMGDr. Walla Al-Hertani is a Medical Bioche...
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