You may qualify for the EMPOWERS clinical research study if you:
  • Are at least 18 years old
  • Have attenuated MPS I
  • 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)

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

Clinical Research FAQ

Here are some common questions and answers about clinical research studies.

A clinical research study is a research project done with human patients to find out if new investigational medications, treatments, or devices are safe, tolerable, and effective. In a clinical research study, the participants get an investigational treatment under the supervision of a doctor and other research professionals. The word “investigational” means it isn’t approved yet by the FDA for general use as a medicine.


By participating, people help doctors find new and improved treatments and better ways to provide care. As a study goes on, the doctor and researchers gather more and more information about the investigational treatment. The study results may show that the investigational treatment improves patient outcomes, offers no benefit to patients, or causes patients unexpected harm. All of these results are important because they advance medical knowledge and help improve patient care.

If regulatory agencies review the study results and feel that the investigational treatment is safe and effective, they may approve it for use by the public.


Each study phase is a different step in the clinical research process, and with each phase the researchers learn more about the treatment.

Phase 1

Studies are the first step in testing new drugs and treatments. In these studies, researchers often give the drug or treatment to a small group of participants to test the safety of different doses, determine how it should be given, and watch closely for any side effects.

Phase 2

Studies usually focus on a particular medical condition. In these studies, researchers give the study drug or treatment to a larger group of participants, including patients with the given medical condition. The researchers watch the participants to see if the treatment is effective, learn more about any side effects, and further test its safety.

Phase 3

Studies are usually the last step before a drug is approved (or not approved) for the public by regulatory agencies. In these studies, researchers give the new treatment or sometimes another commonly used treatment to participants with the given medical condition. Researchers compare the treatments, confirm the new treatment’s effectiveness, monitor its side effects, and collect information that will allow the drug or treatment to be used safely.

Phase 4

Studies are done after the drug or treatment has been approved by regulatory agencies and marketed for public use. These studies continue testing the drug or treatment to collect information about its effect and gather data on any side effects associated with long-term use.


After a clinical research study is finished, all of the information is collected and analyzed to help determine the medication’s safety, effectiveness, and side effects.

Please talk to your doctor or healthcare provider to find your treatment options after you complete a study.


Clinical research studies are regulated, following rules set by the United States Food and Drug Administration. The research study will follow a protocol, which is a detailed study plan explaining what researchers will do in the study.

Each research study must also be approved by an institutional review board (IRB). IRBs are groups of people who help protect the rights and welfare of people participating in research studies. IRBs are usually made up of doctors, scientists, religious representatives, and other medical and nonmedical people.

Your privacy will also be protected. The research team can’t tell anyone that you’re participating in a research study without your permission. All of the information collected during the study will be kept confidential, and your name won’t be listed in any reports based on the study.


Yes, you have the right to leave a research study at any time. If you want to leave, tell the doctor or research team and explain your reasons for leaving. If you decide to leave the EMPOWERS study after receiving the injection, that will mean you’ll forgo monitoring from study doctors and follow-up care.

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