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Found 23 clinical trials
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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 Dec, 2021
  • 4 locations
None
Cardiovascular Structure and Function in the Mucopolysaccharidoses

This study's investigators previously demonstrated the potential utility of non-invasive carotid ultrasonography to calculate carotid intima media thickness (cIMT) and stiffness (as measured by the three parameters, carotid cross-sectional distensibility [cCSD], carotid cross-sectional compliance [cCSC], and carotid incremental elastic modulus [cIEM]) in people with mucopolysaccharidoses (MPS). Investigators also studied arterial …

cytokines
atherosclerosis
gene expression
  • 0 views
  • 08 Oct, 2021
  • 1 location
None
Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

This protocol (GENEFU) provides a mechanism for the 15-year followup period that the FDA requires for all participants in gene transfer protocols and assures that adequate followup can be maintained for a wide variety of participants on different individual gene therapy protocols at St. Jude Children's Research Hospital. GENEFU serves …

second malignancies
late effects
cancer
blood disorder
primary cancer
  • 22 views
  • 27 Jan, 2021
  • 1 location