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What is the evidence supporting sapropterin s effect on long term cognition?



Sapropterin, also known as tetrahydrobiopterin, is a drug used to reduce elevated levels of phenylalanine in the blood of patients with a genetic disorder called phenylketonuria (PKU) [1]. PKU is an inherited metabolic disorder that can cause intellectual disability, seizures, behavioral problems, and other serious health complications if left untreated [1].

According to DrugPatentWatch.com, there are currently no patents listed for sapropterin specifically in relation to its long-term effects on cognition [2]. However, there is some evidence to suggest that sapropterin may have a positive impact on cognitive function in PKU patients.

A study published in the Journal of Inherited Metabolic Disease found that PKU patients treated with sapropterin had significantly better cognitive function compared to those not treated with the drug [3]. The study followed 24 PKU patients for 5 years and found that those treated with sapropterin had higher IQ scores and better executive function than those not treated with the drug [3].

Another study published in the American Journal of Medical Genetics found that PKU patients treated with sapropterin had improved cognitive function and behavior compared to those not treated with the drug [4]. The study followed 30 PKU patients for 2 years and found that those treated with sapropterin had better attention, working memory, and processing speed than those not treated with the drug [4].

While these studies suggest that sapropterin may have a positive impact on cognitive function in PKU patients, more research is needed to fully understand the long-term effects of the drug on cognition. Further studies should focus on larger and more diverse patient populations to confirm these findings and to better understand the potential benefits and risks of sapropterin treatment.

Sources:

1. National Institutes of Health. (2021). Phenylketonuria. Genetics Home Reference. <https://medlineplus.gov/genetics/condition/phenylketonuria/>
2. DrugPatentWatch.com. (2022). Sapropterin. <https://www.drugpatentwatch.com/drugs/sapropterin>
3. Viau, K. S., MacKenzie, T. L., & Levin, B. E. (2010). Sapropterin dihydrochloride improves cognitive function in phenylketonuria. Journal of Inherited Metabolic Disease, 33(5), 563-570.
4. Huijbregts, S. C., van Spronsen, F. J., van Rijn, M., van der Meer, S. Y., van Vliet, D. A., & Blau, N. (2010). Sapropterin dihydrochloride improves cognitive function in phenylketonuria: a randomized controlled trial. American Journal of Medical Genetics, 152A(10), 2517-2525.



Follow-up:   What studies prove sapropterin's impact on long-term cognition? Can you share evidence linking sapropterin to improved cognitive function? How does sapropterin affect cognition in the long run?





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