Methylmalonic Acidemia clinical trials at University of California Health
2 research studies open to eligible people
Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
open to eligible people ages 1 year and up
This is a study of mRNA-3705 in participants with isolated elevated methylmalonic acid (MMA) due to methylmalonyl-coenzyme A (CoA) mutase (MUT) deficiency. The main goal of the study is to assess safety, pharmacokinetics, and pharmacodynamics of mRNA-3705.
at UCLA
Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
open to eligible people ages 1 year and up
The primary objective of this study is to evaluate the long-term safety of mRNA-3705 administered to participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency who have previously participated in other clinical studies of mRNA-3705.
at UCLA
Our lead scientists for Methylmalonic Acidemia research studies include Gerald S. Lipshutz, MD.
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