Hyperammonemia due to N-acetylglutamate synthase deficiency

MONDO:0009377

N-acetylglutamate synthase (NAGS) deficiency is a urea cycle disorder leading to hyperammonaemia.

Also known as: NAGS deficiency, hyperammonemia due to N-acetylglutamate synthase deficiency, N-acetyl glutamate synthetase deficiency, N-acetylglutamate synthase deficiency, N-acetylglutamate synthetase deficiency, NAG synthetase deficiency, NAGSD, Nags deficiency

6 clinical trials for this condition and its sub-types, 2 tagged with Hyperammonemia due to N-acetylglutamate synthase deficiency itself.

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Where it sits in the disease tree

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Part of

↑ Urea cycle disorder or inherited hyperammonemia (24)

Including sub-types (6) Tagged with Hyperammonemia due to N-acetylglutamate synthase deficiency (2)

Not yet recruiting 1 Not yet finished but already full! 1

Can a new injection tame the toxic ammonia of urea cycle disorders?

Disease control Not yet recruiting

This early-stage trial is evaluating an experimental drug called KRRO-121, given as a shot under the skin, to see if it is safe and tolerable. The study first tests KRRO-121 in healthy adults, then in people with urea cycle disorders—a group of inherited conditions that impair th…

Phase 1/2 • Sponsor: Korro Bio, Inc. • Aim: Disease control

Last updated Aug 22, 2026 00:00 UTC

Hyperammonemia due to N-acetylglutamate synthase deficiency

Where it sits in the disease tree

Can a new injection tame the toxic ammonia of urea cycle disorders?