mRNA-3705 MMA Study
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Description
The purpose of this study is to find out more about mRNA-3705, a study drug that is being tested and is not approved by the U.S. Food and Drug Administration (FDA). In this study you will receive mRNA-3705, which is being developed to treat people with isolated MMA with MUT deficiency mRNA-3705. mRNA-3705 can only be used in a study like this one. We want to see what dose of the study drug is safe, how the study drug affects the function of cells, and how much study drug is in the blood after patients receive it.
Eligibility and criteria
What to expect
If you agree to take part, your participation will last about 154 weeks (a little over 3 years) and will involve up to about 69 study visits. Some of these visits may be completed at your home by a Home Health Nurse if you are interested in using this service. There are differences between this study and your usual care. As a participant in this study, you will:
- Receive a study drug via an intravenous (IV) infusion.
- Stay inpatient to monitor reaction to study drug.
- Have research checkups that include testing, blood draws, interviews, and questionnaires.
Related specialties
We need families like you
Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.