TBIO.jpg
Translate Bio Presents Preclinical Data Supporting MRT5201 for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting
08 avr. 2019 16h05 HE | Translate Bio, Inc.
-- MRT5201 was efficiently delivered to the liver of a mouse model of OTC deficiency -- -- Preclinical data demonstrated a single IV dose of MRT5201 was sufficient to protect against hyperammonemia...