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Clinical and Basic Investigations of Methylmalonic Acidemia (MMA) and Related Disorders
Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid metabolism, is heterogeneous in etiology and clinical manifestations. Affect…
Organic AcidemiaMethylmalonic AcidemiaInborn Errors of Metabolism
National Human Genome Research Institute (NHGRI)NCT00078078
A Prospective, Longitudinal, Observational, Multi-centre Study of Pediatric Participants up to 16 Years of Age With Methylmalonyl-CoA Mutase Deficiency Caused by Mutations in the MMUT Gene That Results in a Diagnosis of Isolated Methylmalonic Acidemia (MMA).
Methylmalonic Acidemia (MMA) is a severe and rare condition that affects how the body turns food into energy. In people with MMA, the body is missing or has a …
Methylmalonic Acidemia (MMA)
Genespire SrlNCT07432880
Phase 1
A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
The primary objective of this study is to evaluate the long-term safety and clinical activity of mRNA-3705 administered to participants with isolated methylmal…
Methylmalonic Acidemia
ModernaTX, Inc.NCT05295433
A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
HyperammonemiaMethylmalonic AcidemiaPropionic Acidemia
RECORDATI GROUPNCT05040178