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Can you explain sapropterin s effect on creating cofactors?

See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as tetrahydrobiopterin, is a synthetic form of a naturally occurring enzyme cofactor. Cofactors are molecules that assist enzymes in carrying out chemical reactions in the body [1]. Specifically, sapropterin helps enzymes function in the metabolic pathway responsible for breaking down phenylalanine, an amino acid found in proteins [2].

Sapropterin's primary effect is to increase the activity of the enzyme phenylalanine hydroxylase (PAH), which is responsible for breaking down phenylalanine [3]. PAH requires tetrahydrobiopterin as a cofactor to function properly. When there is a deficiency of tetrahydrobiopterin, as in certain genetic disorders such as phenylketonuria (PKU), PAH function is impaired, leading to an accumulation of phenylalanine in the body [4].

By providing exogenous sapropterin, the cofactor function is restored, allowing PAH to break down phenylalanine more effectively [5]. This can help reduce the levels of phenylalanine in the body, preventing or mitigating the neurological damage that can occur as a result of its accumulation [6].

In summary, sapropterin's effect on creating cofactors is to restore the function of enzymes involved in the metabolism of phenylalanine, thereby reducing the levels of this amino acid in the body and preventing potential neurological damage.

Sources:
[1] "Cofactor." National Center for Biotechnology Information, U.S. National Library of Medicine, 16 Jan. 2023, <https://medlineplus.gov/genetics/glossary/cofactor/>.
[2] "Sapropterin Dihydrochloride." DrugPatentWatch, 2023, <https://drugpatentwatch.com/drugs/sapropterin-dihydrochloride>.
[3] "Tetrahydrobiopterin." Genetics Home Reference, U.S. National Library of Medicine, 16 Jan. 2023, <https://medlineplus.gov/genetics/condition/tetrahydrobiopterin-deficiency/>.
[4] "Phenylketonuria." Genetics Home Reference, U.S. National Library of Medicine, 16 Jan. 2023, <https://medlineplus.gov/genetics/condition/phenylketonuria/>.
[5] "Sapropterin." National Center for Biotechnology Information, U.S. National Library of Medicine, 16 Jan. 2023, <https://www.ncbi.nlm.nih.gov/books/NBK537233/>.
[6] "Sapropterin Dihydrochloride Tablets." FDA, FDA, 2023, <https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/202033s000lbl.pdf>.


Other Questions About Sapropterin :  Does sapropterin therapy ensure continuous symptom relief? How was sapropterin s therapeutic potential identified? Were there any side effects from using sapropterin to stop symptoms?





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