Xuriden (uridine triacetate; Wellstat Therapeutics) has been approved for the treatment of hereditary orotic aciduria, the first approved treatment for this rare metabolic disorder.

Cecilia had hereditary orotic aciduria. "They were very honest, like, we've never had a patient with this," Rebekah Shasky said.

What is glutaric acidemia type 1? Read on to learn more about this metabolic disorder, including its causes, possible symptoms, and treatment options.

Today, the U.S. Food and Drug Administration approved Xuriden (uridine triacetate), the first FDA-approved treatment for patients with hereditary orotic aciduria. Hereditary orotic aciduria is a ...

For the first time, the rare, genetic disease MMA has been studied using a multiomics approach, resulting in an increase in correct diagnoses of patients.

Neven et al. (June 28 issue) 1 discuss the use of bone marrow transplantation in the treatment of severe mevalonic aciduria. We provide additional evidence for the effective cure of mevalonic ...

Proof of concept has been achieved for a therapy for argininosuccinic aciduria consisting of human argininosuccinic lyase (ASL) mRNA encapsulated in lipid nanoparticles (ASL-LNPs).

US regulators have approved Wellstat Therapeutics' uridine triacetate for the treatment of hereditary orotic aciduria (HOA), an ultra-orphan indication that has been reported in only 20 people ...

Argininosuccinic aciduria (ASA) is a genetic disease where an enzymatic protein from the urea cycle pathway is defective in liver cells. Patients with ASA develop intoxication due to ammonia ...