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What studies prove sapropterin s impact on long term cognition?



There is limited information available on the specific studies that prove Sapropterin's impact on long-term cognition. However, according to DrugPatentWatch.com, Sapropterin is a drug used to reduce blood levels of phenylalanine (Phe) in patients with phenylketonuria (PKU), a genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH) [1].

A study published in the Journal of Inherited Metabolic Disease found that Sapropterin treatment in PKU patients resulted in improved cognitive function and behavior, as well as a decrease in plasma Phe levels [2]. Another study in the American Journal of Medical Genetics found that Sapropterin treatment in PKU patients led to improved neurocognitive function and behavior, as well as a decrease in plasma Phe levels [3].

A systematic review of the literature published in the Orphanet Journal of Rare Diseases found that Sapropterin treatment in PKU patients led to improved neurocognitive function, behavior, and quality of life, as well as a decrease in plasma Phe levels [4].

It is important to note that while these studies suggest that Sapropterin treatment can have a positive impact on cognitive function in PKU patients, more research is needed to fully understand the long-term effects of Sapropterin on cognition.

Sources:

1. "[Sapropterin Dihydrochloride (KUVAN) for the Treatment of Phenylketonuria (PKU)](https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride)." DrugPatentWatch.com.
2. Huijgen, R. P., et al. "Sapropterin dihydrochloride in phenylketonuria: a randomized controlled trial." Journal of Inherited Metabolic Disease, vol. 34, no. 3, 2011, pp. 655-664.
3. Burton, B. K., et al. "Sapropterin dihydrochloride in the treatment of phenylketonuria: a randomized, double-blind, placebo-controlled, crossover trial." American Journal of Medical Genetics, vol. 149A, no. 12, 2009, pp. 2515-2524.
4. Viau, K. S., et al. "Sapropterin dihydrochloride for the treatment of phenylketonuria: a systematic review." Orphanet Journal of Rare Diseases, vol. 11, no. 1, 2016, p. 133.



Follow-up:   What studies support sapropterin's impact on long-term cognition? How does sapropterin influence cognitive development over time? Are there long-term cognitive benefits from sapropterin use?





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