Carglumic

Last Updated: 04/28/2026

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Found 74 publications

The role of real-world data and real-world evidence in advancing regulatory science and targeted therapeutics: a narrative review from the United States perspective.

The role of real-world data and real-world evidence in advancing regulatory science and targeted therapeutics: a narrative review from the United States perspective.

Journal: Personalized medicine

Published: April 04, 2026

Carglumic acid and mesalazine as potential anti-mycobacterial agents: a spectroscopic study for repurposing drugs against Mycobacterium tuberculosis targeting its essential enzyme ThyX.

Carglumic acid and mesalazine as potential anti-mycobacterial agents: a spectroscopic study for repurposing drugs against Mycobacterium tuberculosis targeting its essential enzyme ThyX.

Journal: Microbiology spectrum

Published: October 13, 2025

Neonatal Presentation of a Case of Carbonic Anhydrase VA Deficiency.

Neonatal Presentation of a Case of Carbonic Anhydrase VA Deficiency.

Journal: Cureus

Published: August 22, 2025

Sequential therapy with carglumic acid in three cases of organic acidemia crisis

Sequential therapy with carglumic acid in three cases of organic acidemia crisis

Journal: Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics

Published: July 22, 2025

Antenatal and Neonatal Management of Siblings With Carbonic Anhydrase VA Deficiency.

Antenatal and Neonatal Management of Siblings With Carbonic Anhydrase VA Deficiency.

Journal: JIMD reports

Published: July 11, 2025

Case Report: Carglumic acid accelerates ammonia clearance in a neonate with methylmalonic acidemia.

Case Report: Carglumic acid accelerates ammonia clearance in a neonate with methylmalonic acidemia.

Journal: Frontiers in pediatrics

Published: June 21, 2025

Current Treatment Modalities for Urea Cycle Disorders.

Current Treatment Modalities for Urea Cycle Disorders.

Journal: Paediatric drugs

Published: December 16, 2024

Carglumic acid as a treatment for persistent hyperammonemia in carnitine-acylcarnitine translocase deficiency: A case study.

Carglumic acid as a treatment for persistent hyperammonemia in carnitine-acylcarnitine translocase deficiency: A case study.

Journal: Molecular genetics and metabolism reports

Published: November 27, 2024

Trends in drug repurposing: Advancing cardiovascular disease management in geriatric populations.

Trends in drug repurposing: Advancing cardiovascular disease management in geriatric populations.

Journal: Current research in translational medicine

Published: August 27, 2024

Carbamoly-phosphate synthetase 1 (CPS1) deficiency: A tertiary center retrospective cohort study and literature review.

Carbamoly-phosphate synthetase 1 (CPS1) deficiency: A tertiary center retrospective cohort study and literature review.

Journal: Molecular genetics and metabolism reports

Published: August 24, 2024

Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control.

Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control.

Journal: Molecular therapy. Nucleic acids

Published: August 08, 2024

Hyperammonemic encephalopathy requiring hemodialysis in a child with distal renal tubular acidosis.

Hyperammonemic encephalopathy requiring hemodialysis in a child with distal renal tubular acidosis.

Journal: Journal of nephrology

Published: July 16, 2024

Showing 1-12 of 74

Last Updated: 04/28/2026