Sapropterin, also known as tetrahydrobiopterin, is a naturally occurring compound that plays a crucial role in neurodevelopment. It is a cofactor for several enzymes involved in the synthesis of neurotransmitters, such as dopamine, serotonin, and noradrenaline [1]. These neurotransmitters are essential for various brain functions, including mood regulation, cognitive processes, and motor control.
Sapropterin is also the active ingredient in Kuvan, a prescription medication used to reduce blood phenylalanine levels in patients with phenylketonuria (PKU), a genetic disorder characterized by an inability to break down the amino acid phenylalanine [2]. Elevated phenylalanine levels can lead to intellectual disability, seizures, and other neurological problems [3]. By lowering phenylalanine levels, sapropterin can help prevent or mitigate these neurodevelopmental issues in individuals with PKU.
In summary, sapropterin plays a significant role in neurodevelopment by facilitating the synthesis of essential neurotransmitters and helping manage PKU, a genetic disorder that can negatively impact neurodevelopment if left untreated.
Sources:
[1] "Sapropterin Dihydrochloride." DrugPatentWatch.com, <https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride>.
[2] "Kuvan (sapropterin dihydrochloride) tablets, for oral use." DailyMed, <https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=2a9e1e2b-9e3e-4e1a-8a0f-6e6b8a8a3b7d&type=display>.
[3] "Phenylketonuria." Mayo Clinic, 25 Nov. 2021, <https://www.mayoclinic.org/diseases-conditions/phenylketonuria/symptoms-causes/syc-20353994>.