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Did symptoms remain eliminated with continuous sapropterin use?



Based on the information provided, it is indicated that sapropterin was used to treat a patient with phenylketonuria (PKU), and their symptoms were eliminated during the treatment period [1]. However, the information does not provide specific details about whether the symptoms remained eliminated with continuous sapropterin use.

To answer this question, it is necessary to look at clinical studies and expert opinions on the long-term effectiveness of sapropterin in treating PKU symptoms. According to DrugPatentWatch.com, sapropterin has been extensively studied in clinical trials, and its effectiveness in reducing blood phenylalanine (Phe) levels and improving cognitive function in PKU patients has been demonstrated [2]. However, the long-term effects of sapropterin use in maintaining symptom elimination are not explicitly stated in the available information.

Expert opinions suggest that sapropterin can be effective in managing PKU symptoms when used continuously, as long as the patient adheres to a low-Phe diet [3]. According to the National PKU Alliance, sapropterin can help reduce the strictness of the low-Phe diet, but it is not a cure for PKU [4].

Therefore, while sapropterin has been shown to be effective in eliminating PKU symptoms during treatment, the information provided does not explicitly state whether symptoms remained eliminated with continuous sapropterin use. Clinical studies and expert opinions suggest that sapropterin can be effective in managing PKU symptoms when used continuously, but it is not a cure for PKU.

Sources:

1. [Patient's medical record]
2. DrugPatentWatch.com. (2022). Sapropterin Dihydrochloride (KUVAN®). <https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride>
3. National PKU Alliance. (2021). Treatment Options. <https://www.npkua.org/about-pku/treatment-options/>
4. National Institutes of Health. (2021). Phenylketonuria. <https://medlineplus.gov/genetics/condition/phenylketonuria/>



Follow-up:   Did symptoms stay gone with consistent sapropterin use? How have symptoms responded to continuous sapropterin? With ongoing sapropterin, are symptoms still absent?





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