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Pegvaliase, sold under the brand name Palynziq, is a medication used for the treatment of the genetic disease phenylketonuria. [5] [9] [10] It is a phenylalanine (Phe)‑metabolizing enzyme. [5] Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels. [5]
As of 2022, BioMarin has six products on the market, each of which is an orphan drug. [20]Tetrahydrobiopterin (branded as Kuvan) (sapropterin dihydrochloride), a small molecule drug for phenylketonuria, introduced in 2007 as the first medication-based intervention to treat phenylketonuria [21]
Valoctocogene roxaparvovec is a gene therapy that uses an adeno-associated virus 5 (AAV5) that codes for human Factor VIII, together with a human liver-specific promoter that encourages translation in hepatocytes, not liver endothelial and sinusoidal cells, where Factor VIII is ordinarily synthesised.
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He was born on 23 August 1888 at Kvam (in the present-day Steinkjer Municipality in Trøndelag county, Norway). Følling studied chemistry at the Norwegian Institute of Technology in Trondheim and graduated in 1916.
Polyethylene glycol. PEGylation (or pegylation) is the process of both covalent and non-covalent attachment or amalgamation of polyethylene glycol (PEG, in pharmacy called macrogol) polymer chains to molecules and macrostructures, such as a drug, therapeutic protein or vesicle, which is then described as PEGylated.
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Phenylalanine ammonia lyase is specific for L-phenylalanine, and to a lesser extent, L-tyrosine. [9] [10] The reaction catalyzed by PAL is a spontaneous elimination reaction rather than an oxidative deamination.