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MS
UMPS AR unreviewed

Hereditary orotic aciduria

Enzyme or protein defect

UMP synthase (OPRT + OMP decarboxylase)

Accumulates or becomes deficient

Orotic acid

Key features

Megaloblastic anaemia NOT responsive to B12 or folate, failure to thrive; NO hyperammonaemia (unlike OTC deficiency)

Management

Oral uridine

Not clinical guidance. Written from standard clinical biochemistry, not from the uploaded documents, and marked unreviewed. Teaching-level reference for study and database seeding only. It must not be used to manage a patient.

Recent literature

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