AI Article Synopsis
- Trofinetide (DAYBUE™) is a synthetic oral medication designed to treat rare childhood neurodevelopmental disorders, specifically Rett syndrome.
- It is based on a modified version of a tripeptide related to insulin-like growth factor-1 (IGF-1) and is developed by Neuren Pharmaceuticals and Acadia Pharmaceuticals.
- The drug received approval in March 2023 in the USA for use in patients aged 2 years and older, marking a significant milestone in its development journey.
Article Abstract
Trofinetide (DAYBUE™), an oral, small molecule, synthetic analog of glycine-proline-glutamate [GPE; the N-terminal tripeptide derivative of insulin like growth factor-1 (IGF-1)], is being developed by Neuren Pharmaceuticals and Acadia Pharmaceuticals for the treatment of rare childhood neurodevelopmental disorders. Trofinetide was approved in March 2023 in the USA for the treatment of Rett syndrome in adult and pediatric patients 2 years of age and older. This article summarizes the milestones in the development of trofinetide leading to this first approval for Rett syndrome.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1007/s40265-023-01883-8 | DOI Listing |
Publication Analysis
Top Keywords
rett syndrome
8
trofinetide
4
trofinetide approval
4
approval trofinetide
4
trofinetide daybue™
4
daybue™ oral
4
oral small
4
small molecule
4
molecule synthetic
4
synthetic analog
4
Similar Publications
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!