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Found 3 clinical trials
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RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine

  • 8 views
  • 03 Oct, 2021
  • 2 locations
None
RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a safety and dose ranging study to

  • 275 views
  • 16 Aug, 2021
  • 5 locations
None
Mucopolysaccharidosis Type II Observational

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 …

  • 0 views
  • 03 Oct, 2021
  • 4 locations