Glycerol phenylbutyrate efficacy and safety from an open label study in pediatric patients under 2 months of age with urea cycle disorders

Suggested guidelines for the diagnosis and management of urea cycle disorders: First revision

(2019) Johannes Häberle et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Progress and challenges in development of new therapies for urea cycle disorders

(2019) Leandro R Soria et al.   HUMAN MOLECULAR GENETICS

Long-term safety and efficacy of glycerol phenylbutyrate for the management of urea cycle disorder patients

(2019) George A. Diaz et al.   MOLECULAR GENETICS AND METABOLISM

Neuropsychological attributes of urea cycle disorders: A systematic review of the literature

(2019) Susan E. Waisbren et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Newborn screening for proximal urea cycle disorders: Current evidence supporting recommendations for newborn screening

(2018) J. Lawrence Merritt et al.   MOLECULAR GENETICS AND METABOLISM

A randomized trial to study the comparative efficacy of phenylbutyrate and benzoate on nitrogen excretion and ureagenesis in healthy volunteers

(2017) Sandesh C S Nagamani et al.   GENETICS IN MEDICINE

Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2 months to 2 years

(2017) Susan A. Berry et al.   MOLECULAR GENETICS AND METABOLISM

Glycerol phenylbutyrate for the maintenance treatment of patients with deficiencies in enzymes of the urea cycle

(2017) Nicola Longo et al.   Expert Opinion on Orphan Drugs

An update on the use of benzoate, phenylacetate and phenylbutyrate ammonia scavengers for interrogating and modifying liver nitrogen metabolism and its implications in urea cycle disorders and liver disease

(2016) Javier De Las Heras et al.   Expert Opinion on Drug Metabolism & Toxicology

Liver Transplantation for Urea Cycle Disorders: Analysis of the United Network for Organ Sharing Database

(2015) L. Yu et al.   TRANSPLANTATION PROCEEDINGS

A longitudinal study of urea cycle disorders

(2014) Mark L. Batshaw et al.   MOLECULAR GENETICS AND METABOLISM

Glycerol phenylbutyrate treatment in children with urea cycle disorders: Pooled analysis of short and long-term ammonia control and outcomes

(2014) Susan A. Berry et al.   MOLECULAR GENETICS AND METABOLISM

Population Pharmacokinetic Modeling and Dosing Simulations of Nitrogen-Scavenging Compounds: Disposition of Glycerol Phenylbutyrate and Sodium Phenylbutyrate in Adult and Pediatric Patients with Urea Cycle Disorders

(2013) Jon P. R. Monteleone et al.   JOURNAL OF CLINICAL PHARMACOLOGY

Ammonia Control in Children Ages 2 Months through 5 Years with Urea Cycle Disorders: Comparison of Sodium Phenylbutyrate and Glycerol Phenylbutyrate

(2013) Wendy Smith et al.   JOURNAL OF PEDIATRICS

Elevated phenylacetic acid levels do not correlate with adverse events in patients with urea cycle disorders or hepatic encephalopathy and can be predicted based on the plasma PAA to PAGN ratio

(2013) M. Mokhtarani et al.   MOLECULAR GENETICS AND METABOLISM

Rare disease clinical research network's urea cycle consortium delivers a successful clinical trial to improve alternate pathway therapy

(2012) Pramod K. Mistry   HEPATOLOGY

Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate

(2012) George A. Diaz et al.   HEPATOLOGY

Ammonia toxicity to the brain

(2012) Olivier Braissant et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Ammonia control in children with urea cycle disorders (UCDs); Phase 2 comparison of sodium phenylbutyrate and glycerol phenylbutyrate

(2011) Uta Lichter-Konecki et al.   MOLECULAR GENETICS AND METABOLISM

Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis

(2010) Brendan M. McGuire et al.   HEPATOLOGY

Alternative pathway or diversion therapy for urea cycle disorders now and in the future

(2010) Stephen Cederbaum et al.   MOLECULAR GENETICS AND METABOLISM

Nitrogen sparing therapy revisited 2009

(2010) Gregory M. Enns   MOLECULAR GENETICS AND METABOLISM

Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: Safety, pharmacokinetics and ammonia control

(2010) Brendan Lee et al.   MOLECULAR GENETICS AND METABOLISM