Hepatic Lactate Dehydrogenase A: An RNA Interference Target for the Treatment of All Known Types of Primary Hyperoxaluria
Gema Ariceta, Kelly Barrios, Bob D. Brown, Bernd Höppe, R. Roßkamp, Craig B. Langman
Abstract
INTRODUCTION: is mainly expressed in the liver and muscles. METHODS: inhibition. RESULTS: Based on a literature review of human LDHA deficiency, we defined the phenotype as mainly muscle-related with no liver manifestations. Healthy volunteers treated with nedosiran experienced no drug-related musculoskeletal adverse events. There were no significant alterations in plasma lactate, pyruvate, or creatine kinase levels in the nedosiran group compared with the placebo group, signaling the uninterrupted interconversion of lactate and pyruvate and normal muscle function. CONCLUSION: inhibition is a safe therapeutic mechanism for the treatment of all known types of PH.