See the DrugPatentWatch profile for sapropterin

Based on the information available, I was unable to find specific patient cases from Sapropterin trials. However, I can provide some general information about Sapropterin and its trials.

Sapropterin, also known as Kuvan, is a prescription medication used to reduce the symptoms of phenylketonuria (PKU) in patients who have a specific enzyme deficiency. PKU is a genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase, which is needed to break down the amino acid phenylalanine. If left untreated, PKU can lead to intellectual disability, seizures, behavioral problems, and other serious health issues.

Clinical trials of Sapropterin have been conducted to evaluate its safety and efficacy in treating PKU. According to the drug's prescribing information, the efficacy of Sapropterin was established in three clinical trials that enrolled a total of 135 patients with PKU. In these trials, patients were randomized to receive either Sapropterin or a placebo, and their blood phenylalanine levels were measured over a period of time.

The results of these trials showed that Sapropterin was effective in reducing blood phenylalanine levels in patients with PKU. In one trial, patients who received Sapropterin had a mean decrease in blood phenylalanine levels of 31% compared to a 6% increase in patients who received a placebo. In another trial, patients who received Sapropterin had a mean decrease in blood phenylalanine levels of 25% compared to a 1% increase in patients who received a placebo.

It is important to note that while these trials provide evidence of Sapropterin's efficacy in reducing blood phenylalanine levels in patients with PKU, they do not provide specific patient cases.

Sources:

1. Kuvan (sapropterin dihydrochloride) tablets for oral use [prescribing information]. BioMarin Pharmaceutical Inc.; 2022. <https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/021963s030lbl.pdf>
2. ClinicalTrials.gov. Sapropterin Dihydrochloride in the Treatment of Phenylketonuria (PKU) [Internet]. Bethesda (MD): National Library of Medicine (US); 2000-[cited 2023 Mar 29]. Available from: <https://clinicaltrials.gov/ct2/show/NCT00069750>
3. DrugPatentWatch. Sapropterin Dihydrochloride (Kuvan) [Internet]. DrugPatentWatch; [cited 2023 Mar 29]. Available from: <https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride-kuvan>