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The trial proceeded without reported toxicity, and no lethal dose threshold (LD1) was ever established in subsequent safety testing meaning researchers administering the compound could not find a dose that caused death in even 1% of test subjects across the animal model safety work that preceded and followed the human study
Deamidation enables pathogenic SMAD6 variants to activate the BMP signaling pathway
Rossiello F, Jurk D, Passos JF, dAdda di Fagagna F
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