Hyperammonemia due to N-acetylglutamate synthase deficiency Advocacy Hub | UniteRare

At a Glance

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1FDA treatments

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Regulatory Watchboard

No FDA or trial events recorded yet for Hyperammonemia due to N-acetylglutamate synthase deficiency.

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

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Patient Assistance Programs1

Carbaglu

Recordati Rare Diseases

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Approved Treatments

1 FDA-approved

Carbaglu

(CARGLUMIC ACID)Orphan drugstandard

Recordati Rare Diseases

Carbamoyl Phosphate Synthetase 1 Activator [EPC]

12.1 Mechanism of Action Carglumic acid is a synthetic structural analogue of N-acetylglutamate (NAG) which is produced from glutamate and acetyl-CoA ...

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Clinical Trial Landscape

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Recent News & Research

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Specialist Network

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