A5102993348- Metabolism and Genetic Disorders
- Diet and metabolism studies
- Mitochondrial Function and Pathology
- Drug Transport and Resistance Mechanisms
- Antibiotics Pharmacokinetics and Efficacy
- Amino Acid Enzymes and Metabolism
- Renal Transplantation Outcomes and Treatments
- Pharmacological Effects and Toxicity Studies
- Curcumin's Biomedical Applications
- Electrolyte and hormonal disorders
- Adrenal Hormones and Disorders
- Neonatal Health and Biochemistry
- Methemoglobinemia and Tumor Lysis Syndrome
- Clinical Nutrition and Gastroenterology
- Pharmacogenetics and Drug Metabolism
PTC Therapeutics (United States)
2021-2025
University of California, San Francisco
1984
Mount Desert Island Biological Laboratory
1984
Yale University
1984
Sepiapterin is an exogenously synthesized new chemical entity that structurally equivalent to endogenous sepiapterin, a biological precursor of tetrahydrobiopterin (BH4), which cofactor for phenylalanine hydroxylase. being developed the treatment hyperphenylalaninemia in pediatric and adult patients with phenylketonuria (PKU). This study employed concentration-QT interval analysis assess QT prolongation risk following sepiapterin treatment. Data from three phase 1 studies one 3 were pooled...
We investigated the role of serum albumin in hepatic uptake organic anions by determining effect added bovine on sulfobromophthalein (BSP) skates, an animal which naturally lacks this protein. A single-pass perfused liver model was used to determine steady-state net rates as BSP or and concentrations were systematically varied. Results indicated that two different steps process are capable limiting rate depending selected. At higher corresponding (up 112 μM ), saturation kinetics observed...
PTC923 (formerly CNSA-001), an oral formulation of sepiapterin, a natural precursor intracellular tetrahydrobiopterin (BH4), has been shown in humans to induce larger increases circulating BH4 vs. sapropterin dihydrochloride. Sapropterin reduces blood phenylalanine (Phe) by ≥20-30% minority subjects with PKU. This was Phase 2 randomized, multicenter, three-period crossover, open-label, active controlled, all-comers [regardless hydroxylase (PAH) variants] comparison 60 mg/kg, 20 mg/kg and 24...
Frequent blood phenylalanine (Phe) measurement is required for phenylketonuria (PKU) patients diagnosis and disease status monitoring. Though various methods are available Phe measurement, there a lack of validated quantitative measuring with less than 15% variability. A method to allow at home sample collection the PKU community in high demand.A volumetric absorptive microsampling (VAMS) dried high-performance liquid chromatography-tandem mass spectrometry (HPLC-MS/MS) was developed fully...
Sepiapterin, also known as PTC923 and CNSA-001, is a synthetic form of endogenous sepiapterin being developed novel oral treatment for phenylketonuria. Sepiapterin natural precursor tetrahydrobiopterin (BH
Sepiapterin is a natural precursor of tetrahydrobiopterin (BH
Abstract Sepiapterin is an orally administered drug in development for the treatment of phenylketonuria, inborn error metabolism characterized by deficiency phenylalanine‐metabolizing enzyme phenylalanine hydroxylase. This study pharmacokinetics, safety, and tolerability 2 clinical sepiapterin formulations (Phase 1/2, Phase 3) effects food on pharmacokinetics 3 formulation healthy participants. In Part A, 18 participants were randomized to one sequences, each with 4 dosing periods comprising...
Tetrahydrobiopterin (BH4) is a cofactor for the enzymes tyrosine hydroxylase and tryptophan hydroxylase, rate-limiting in production of neurotransmitters, dopamine serotonin, respectively, central nervous system (CNS). Administration BH4 used clinically within management persons with genetic deficiencies, but molecule does not cross blood-brain barrier sufficiently. CNSA-001 pharmaceutical preparation sepiapterin, natural precursor that induced larger increases plasma compared administration...