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. clinicaltrials.gov.
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 firstname.lastname@example.org.