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trofinetide (Daybue)
Indications:
- treatment of Rett syndrome in adults & children >= 2 years
Dosage:
- twice a day oral dosing
Adverse effects:
- diarrhea, vomiting (most common)
Mechanism of action:
- synthetic analog of the amino-terminal tripeptide of IGF-1, which occurs naturally in the brain
- potentially reduces neuroinflammation & supports synaptic function
General
heterocyclic compound, 1 ring
amide
carboxylate
amine
neurologic agent
Database Correlations
PUBCHEM cid=11318905
References
- Brooks M
FDA OKs First Drug for Rett Syndrome
Medscape. March 14, 2023
https://www.medscape.com/viewarticle/989649