Accelerating Drug Repurposing for Rett Syndrome Project
Publication Date
4-25-2025
Abstract
Rett Syndrome is a rare, debilitating genetic disorder that has very limited treatment options. Rett primarily affects females and is caused by a mutation in a gene (MECP2) that plays a critical role in neurodevelopment. Children born with Rett appear to reach their milestones in infancy, but however eventually start regressing and losing ability to speak, as well as loss of motor skills, seizures, and repetitive hand movements. Many families and researchers are continuously searching for new treatments and cures for Rett, however the challenge is efficiently identifying which drugs are mechanistically suitable for Rett, as well as safe to use for the Rett population. Furthermore, time is limited, and drug discovery in general takes at least twelve years. With the combination of a rapid rise of artificial intelligence and new innovative databases such as PharmAlchemy, drug discovery and repurposing could potentially be accelerated, giving the next generation of individuals with rare disorders a potential, quicker cure.
Keywords
drug repurposing, info603, rett syndrome
Repository
Zenodo
Repository Identifier
https://zenodo.org/records/15278272
Distribution License
This work is licensed under a Creative Commons Attribution 4.0 International License.
