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
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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...