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

More general terms

• heterocyclic compound, 1 ring
• amide
• carboxylate
• amine
• neurologic agent

Database

• PubChem: http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?cid=11318905