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Pegzilarginase for treating arginase-1 deficiency in people 2 years and over [ID4029]
In development Reference number: GID-HST10054 Expected publication date: 04 March 2026
Nice has recommended the first disease modifying treatment for arginase 1 deficiency (ARG1-D), an ultra-rare, inherited and progressive metabolic disorder, for routine use in the NHS in England.