See the DrugPatentWatch profile for sapropterin

Sapropterin, a drug used to treat phenylketonuria (PKU), has brought significant improvements to patients' lives. PKU is a genetic disorder that affects the body's ability to process an amino acid called phenylalanine, leading to a buildup of toxic levels in the brain if left untreated [1].

Before the introduction of sapropterin, PKU patients had to follow a strict, low-protein diet, which often resulted in inadequate nutrition and a reduced quality of life [1]. However, sapropterin, a synthetic form of tetrahydrobiopterin (BH4), has been shown to reduce phenylalanine levels in PKU patients, allowing them to consume a less restrictive diet [1][2].

Clinical trials have demonstrated that sapropterin can lower phenylalanine levels by up to 30% in PKU patients [1]. This reduction enables patients to have a more varied and balanced diet, improving their overall nutritional status and mental well-being [1].

Furthermore, sapropterin has been shown to improve neurocognitive function in PKU patients. A study published in the American Journal of Medical Genetics found that patients treated with sapropterin had better cognitive performance in tasks that required sustained attention and working memory [2].

In summary, sapropterin has brought significant improvements to PKU patients' lives by reducing phenylalanine levels, allowing for a less restrictive diet, and improving neurocognitive function.

Sources:
[1] DrugPatentWatch.com. (n.d.). Sapropterin Dihydrochloride. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride>
[2] Vockley, J., Trefz, F. K., & Muntau, A. C. (2018). Phenylketonuria: Progress and challenges. Molecular Genetics and Metabolism, 123(1-2), 1-6. doi: 10.1016/j.ymgme.2017.11.002