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Organic Acidemia
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Organic Acidemia trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Organic Acidemia trials you may qualify forBackground: People s bodies need to break down food into the chemicals. These chemicals are used for energy and growth. Some people cannot process all chemical…
Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid metabolism, is heterogeneous in etiology and clinical manifestations. Affecte…
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously pa…
This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to d…
Ammonia is a waste product of protein and amino acid catabolism and is also a potent neurotoxin. High blood ammonia levels on the brain can manifest as cytotoxi…
This is a prospective mixed-design study focused on the long-term management of propionic aciduria (PA) and methylmalonic aciduria (MMA) with N-carbamylglutamat…
Background: \- People with inborn errors of metabolism can t turn food into energy the right way. This can affect a person s growth and health. Researchers wan…
The goal of this observational study is to learn if people with certain ammonia metabolism disorders will measure their ammonia levels at home. The main questi…
Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)…