mRNA-3927 for Propionic Acidemia Study
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Description
The purpose of this study is to find out more about an investigational drug called mRNA-3927 (the study drug). An "investigational drug" is a drug that is being tested and is not approved by the U.S. Food and Drug Administration (FDA). We want to see how safe the study drug is and if it works for participants with propionic acidemia (PA).
Eligibility and criteria
What to expect
If you agree to take part, your participation will last about 1.5 years and will involve approximately 44 study visits, some of which may be completed by a Home Health Nurse should you be interested in using this service. There are differences between this study and your usual care. As a participant in this research, you will:
- Receive a study drug via an intravenous (IV) infusion.
- Stay inpatient to monitor reaction to study drug.
- Have research blood draws, EKGs, Echos, and complete questionnaires.
Related specialties
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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.