Inborn organic aciduria
MONDO:0000688An inherited disorder that affects the metabolism of any acidic compound containing carbon in a covalent linkage.
Also known as: disorder of organic acid metabolism, organic acid metabolism disorder, inborn error of organic acid metabolic process, inborn organic acid metabolic process disorder, organic acidemia, organic aciduria, rare inborn error of organic acid metabolic process, inherited organic acidemia
29 clinical trials for this condition and its sub-types.
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Broader categories
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Gene therapy for rare metabolic disease passes early safety check
Disease control OngoingThis study follows 4 people with methylmalonic acidemia who previously received hLB-001 gene therapy. Researchers are checking for long-term side effects. The goal is to see if the treatment remains safe over time.
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:02 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC