Efficacy and Safety of Pegzilarginase in Arginase 1 Deficiency (PEACE): a Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Centre Trial
ECLINICALMEDICINE（2024）
Key words
Arginine,Arginase 1,ARG1-D,Enzyme therapy,Gross motor function measure,Guanidino compounds,Hyperargininaemia,Inherited metabolic disorder,Inborn error of metabolism,Mobility,Novel therapeutics,Rare disease,Spasticity,Timed walk test,Urea cycle disorder
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