See the DrugPatentWatch profile for sapropterin
Sapropterin, also known as tetrahydrobiopterin, is a drug used to reduce elevated phenylalanine levels in the blood of individuals with a genetic disorder called phenylketonuria (PKU) [1]. PKU is an inherited metabolic disorder that affects the body's ability to process phenylalanine, an amino acid found in proteins [1].
According to DrugPatentWatch.com, sapropterin has been studied in specific patient populations within PKU research [2]. These patient populations include:
1. Pediatric patients: Sapropterin has been studied in pediatric patients with PKU to evaluate its safety and efficacy in reducing phenylalanine levels [3].
2. Adult patients: Clinical trials have been conducted to assess the long-term safety and efficacy of sapropterin in adult patients with PKU [4].
3. Pregnant women with PKU: Sapropterin has been investigated in pregnant women with PKU to evaluate its safety and efficacy in managing phenylalanine levels during pregnancy [5].
4. Patients with mild PKU or non-PKU hyperphenylalaninemia: Sapropterin has been studied in patients with mild PKU or non-PKU hyperphenylalaninemia to determine its potential benefits in these populations [6].
It is important to note that while sapropterin has been studied in these specific patient populations, its use should always be under the guidance and supervision of a healthcare professional.
Sources:
[1] "Sapropterin dihydrochloride." National Center for Biotechnology Information, U.S. National Library of Medicine, <
https://pubchem.ncbi.nlm.nih.gov/compound/Sapropterin-dihydrochloride>.
[2] "Sapropterin (Brand Name: Kuvan) Drug Profile." DrugPatentWatch.com, <
https://www.drugpatentwatch.com/drugs/sapropterin>.
[3] "Safety and Efficacy of Sapropterin Dihydrochloride in Pediatric Patients With Phenylketonuria." ClinicalTrials.gov, U.S. National Library of Medicine, <
https://clinicaltrials.gov/ct2/show/NCT00291227>.
[4] "Long-Term Safety and Efficacy of Sapropterin Dihydrochloride in Adult Patients With Phenylketonuria." ClinicalTrials.gov, U.S. National Library of Medicine, <
https://clinicaltrials.gov/ct2/show/NCT00291214>.
[5] "Safety and Efficacy of Sapropterin Dihydrochloride in Pregnant Women With Hyperphenylalaninemia." ClinicalTrials.gov, U.S. National Library of Medicine, <
https://clinicaltrials.gov/ct2/show/NCT01069803>.
[6] "Efficacy and Safety of Sapropterin Dihydrochloride in Patients With Mild Hyperphenylalaninemia." ClinicalTrials.gov, U.S. National Library of Medicine, <
https://clinicaltrials.gov/ct2/show/NCT01435154>.