Browse Studies — Arctuva

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

Estimated

~$10 – $675

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)

Estimated

~$10 – $1,125

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

Estimated

~$10 – $8,750

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

Estimated

~$10 – $1,125

RECORDATI GROUPNCT05040178