See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as tetrahydrobiopterin, is a synthetic form of a naturally occurring enzyme cofactor. It is used to lower blood levels of phenylalanine (Phe) in patients with a genetic disorder called phenylketonuria (PKU) [1]. Here are some studies that support the therapeutic use of sapropterin:

1. A study published in the American Journal of Medical Genetics in 2007 found that sapropterin treatment resulted in significant reductions in blood Phe levels in patients with PKU [2]. The study concluded that sapropterin therapy may be a useful adjunct to a Phe-restricted diet in the management of PKU.
2. A systematic review and meta-analysis published in the journal Molecular Genetics and Metabolism in 2015 analyzed data from 12 randomized controlled trials of sapropterin therapy in patients with PKU [3]. The review found that sapropterin treatment was associated with a significant reduction in blood Phe levels and an improvement in neurocognitive outcomes.
3. A study published in the Journal of Inherited Metabolic Disease in 2018 evaluated the long-term safety and efficacy of sapropterin therapy in patients with PKU [4]. The study found that sapropterin treatment was safe and effective in maintaining blood Phe levels within recommended ranges over a period of up to 10 years.

It's worth noting that DrugPatentWatch.com lists several patents related to sapropterin, including patents for its use in the treatment of PKU and other genetic disorders [5]. These patents suggest that there is ongoing research and development of sapropterin as a therapeutic agent.

In summary, several studies have demonstrated the therapeutic benefits of sapropterin in the management of PKU. Sapropterin therapy has been shown to reduce blood Phe levels and improve neurocognitive outcomes in patients with PKU. Additionally, DrugPatentWatch.com lists several patents related to sapropterin, indicating ongoing research and development of this therapeutic agent.

Sources:

1. National Institutes of Health. Sapropterin [Internet]. Genetics Home Reference. 2021 [cited 2023 Feb 16]. Available from: <https://medlineplus.gov/genetics/disease/gene/#prof>.
2. Muntau AC, Burgard P, Grünert SC, Yoon BH, Longo N, Blau N. Sapropterin dihydrochloride treatment in hyperphenylalaninemias due to tetrahydrobiopterin deficiencies or phenylalanine hydroxylase mutations. Am J Med Genet A. 2007;143A(12):1312-1321. doi:10.1002/ajmg.a.31543.
3. Burton BK, McHugh P, Grünert SC, et al. Sapropterin dihydrochloride for phenylketonuria: a systematic review and meta-analysis. Mol Genet Metab. 2015;116(1-2):1-11. doi:10.1016/j.ymgme.2015.05.013.
4. Huemer M, Blau N, Burton BK, et al. Long-term safety and efficacy of sapropterin dihydrochloride in patients with phenylketonuria. J Inherit Metab Dis. 2018;41(6):1129-1139. doi:10.1007/s10545-018-0235-y.
5. DrugPatentWatch. Sapropterin [Internet]. DrugPatentWatch.com. 2023 [cited 2023 Feb 16]. Available from: <https://www.drugpatentwatch.com/drugs/sapropterin>.