ClinicaliQ Trial Snapshot
- A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene — Recruiting • Gastroenterology • NCT07432880.
- Study Summary for UK Clinicians.
- This observational study tracks children with methylmalonic acidemia to identify reliable blood markers and clinical signs indicating disease activity for future treatment development.
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What This Trial Is Studying
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 very low activity of a specific protein (an enzyme called methylmalonyl-CoA mutase (MMUT)) needed to break down certain proteins and fats in everyday food. Because this process does not work properly, a harmful substance called methylmalonic acid builds up in the blood and tissues, causing damage in the body. Most people with MMA have an altered MMUT gene, which affects the…