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Research Studies

Metabolic Disease Program Research Studies

Find research studies available to children cared for by the Metabolic Disease Program team.

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mRNA-3705 MMA Study

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.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

mRNA-3927 for Propionic Acidemia Study

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).

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

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