Living with MPS II?

Talk to your doctor or nurse about the CHAMPIONS study.

To learn more about the study opportunity, please contact Contact Us

Mucopolysaccharidosis II (MPS II) is a genetic disorder in which people’s bodies are missing an enzyme named iduronate 2-sulfatase. This enzyme is needed to properly break down different types of large sugar molecules called glycosaminoglycans, or GAGs.

Doctors are studying SB-913, a type of investigational gene therapy called genome editing. Doctors want to find out whether or not it is an effective, lasting treatment that will make your body able to break down GAGs. The CHAMPIONS study will test SB-913 to see if it’s safe and tolerable. “Investigational” means it isn’t approved by the FDA for general use as a medicine.

Please have a look around the site for more information about this study and gene therapy.

You may qualify for the CHAMPIONS clinical research study if you: 

  • Are at least 5 years old
  • Have MPS II
  • Are able and willing to use corticosteroids

People who qualify will receive:

  • Investigational gene therapy (SB-913) for MPS II
  • Study-related medical care
  • Reimbursement for travel (transportation assistance may be available)

To learn more about the study opportunity, please contact

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