Living with MPS I?

You could qualify for the EMPOWERS study.

Mucopolysaccharidosis I (MPS I) is a genetic disorder. People’s bodies need to break down different types of large sugar molecules, called “glycosaminoglycans” (or “GAGs”). People with MPS I are missing the α-L-iduronidase (IDUA) enzyme, which is needed to break down GAGs.

Doctors are studying SB-318, a type of investigational gene therapy called genome editing. (“Investigational” means it isn’t approved by the FDA for general use as a medicine.) 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 EMPOWERS study will test SB-318 to see if it’s safe and tolerable.

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

  • Are at least 18 years old
  • Have attenuated MPS I
  • Have MPS I treated by bone marrow transplant
  • Are able and willing to use corticosteroids

People who qualify will receive:

  • Investigational gene therapy (SB-318) for MPS I
  • 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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