See the DrugPatentWatch profile for sapropterin
Sapropterin, also known as tetrahydrobiopterin, is a medication used to reduce the symptoms of phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine. [1]
According to the available information, sapropterin can have a significant impact on several aspects of well-being for individuals with PKU. Specifically, it can help to lower the levels of phenylalanine in the blood, which can lead to improvements in neuropsychological functioning, including cognitive and behavioral symptoms. [2]
A study published in the American Journal of Medical Genetics found that sapropterin treatment was associated with significant improvements in several areas of neuropsychological functioning, including attention, working memory, and executive function. [3] Additionally, the study found that sapropterin treatment was also associated with improvements in behavior, including reduced hyperactivity and irritability.
Another study published in the Journal of Inherited Metabolic Disease found that sapropterin treatment was associated with significant reductions in phenylalanine levels, which led to improvements in cognitive function and behavior. [4]
It's important to note that while sapropterin can have a positive impact on well-being for individuals with PKU, it is not a cure for the disorder. Additionally, not all individuals with PKU respond to sapropterin treatment, and it is important for individuals to work closely with their healthcare providers to determine the most effective treatment plan for their specific needs. [5]
In summary, sapropterin can have a significant impact on several aspects of well-being for individuals with PKU, including neuropsychological functioning and behavior. However, it is not a cure for the disorder and not all individuals with PKU respond to sapropterin treatment.
Sources:
1. <
https://www.drugpatentwatch.com/drugs/sapropterin>
2. <
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5842353/>
3. <
https://onlinelibrary.wiley.com/doi/abs/10.1002/ajmg.a.32239>
4. <
https://onlinelibrary.wiley.com/doi/abs/10.1002/jimd.29873>
5. <
https://rarediseases.org/rare-diseases/phenylketonuria/>