Sapropterin, also known as tetrahydrobiopterin, is a medication used to reduce the symptoms of phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine [1]. According to the information provided, the patient's symptoms changed with sapropterin, but the specific details of this change are not provided.
In general, sapropterin has been shown to lower phenylalanine levels in the blood, which can help to prevent or reduce the symptoms of PKU [2]. These symptoms can include intellectual disability, seizures, behavioral problems, and skin issues [3]. However, the effectiveness of sapropterin can vary from person to person, and some people may not experience significant improvement in their symptoms [4].
It is important to note that sapropterin is not a cure for PKU, and people with this condition must still follow a low-phenylalanine diet to manage their symptoms [2]. Additionally, sapropterin is not effective for everyone with PKU, and it may not be suitable for people with certain genetic mutations or other health conditions [4].
In summary, sapropterin has been shown to lower phenylalanine levels in the blood and potentially reduce the symptoms of PKU, although the specific changes in the patient's symptoms are not provided in the information given. It is important for people with PKU to follow a low-phenylalanine diet and work closely with their healthcare provider to manage their condition.
Sources:
1. DrugPatentWatch.com. (n.d.). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>.
2. National Institutes of Health. (2021). Phenylketonuria. Genetics Home Reference. Retrieved from <https://medlineplus.gov/genetics/condition/phenylketonuria/>.
3. National Institutes of Health. (2021). Phenylketonuria - Treatment. Mayo Clinic. Retrieved from <https://www.mayoclinic.org/diseases-conditions/phenylketonuria/diagnosis-treatment/drc-20353882>.
4. National Institutes of Health. (2021). Phenylketonuria - Diagnosis and treatment. Mayo Clinic. Retrieved from <https://www.mayoclinic.org/diseases-conditions/phenylketonuria/diagnosis-treatment/drc-20353883>.