Daybue,the first new drug for the treatment of Rett syndrome
Daybue oral solution has been approved by the U.S.Food and Drug Administration(FDA)in March 2023 for the treatment of Rett syndrome in adults and children aged two years and above,becoming the first and only drug approved for the treatment of Rett syndrome.The main active component of Daybue is trofinetide,a novel synthetic analogue of the amino terminal tripeptide of insulin-like growth factor Ⅰ(IGF-1).It can stimulate synaptic maturation,overcome synaptic and neuronal immaturity,and treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function.This paper introduces it's mechanism of action,pharmacodynamics,pharmacokinetics,clinical research and safety.
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