Translational Pharmacokinetic/Pharmacodynamic Model for mRNA-3927, an Investigational Therapeutic for the Treatment of Propionic Acidemia

Attarwala, H; Lumley, M; Liang, M; Ivaturi, V; Senn, J

Liang, M (通讯作者),Moderna Inc, Technol Sq 200, Cambridge, MA 02139 USA.

NUCLEIC ACID THERAPEUTICS, 2023; 33 (2): 141

Abstract

Propionic acidemia (PA) is an ultrarare disorder caused by deficiency of the mitochondrial enzyme, propionyl-CoA carboxylase (PCC), composed of PCCA a......

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