Development of an Artificial Intelligence/Machine Learning (AI/ML) model-based clinical trial simulation platform to better characterize Loss of Ambulation in Duchenne Muscular Dystrophy (DMD)

This project aims to develop a new technology to build tools that improve drug development for Duchenne Muscular Dystrophy (DMD). DMD is a rare genetic condition that affects about 1 in every 3,500 male births worldwide. It causes muscle weakness and progressive loss of muscle function, leading to severe disability and significantly shortened life expectancy.

Currently, developing treatments for DMD is challenging, time-consuming, and expensive. This research seeks to address these challenges by creating tools that better predict how DMD progresses over time. These tools will also help researchers understand how safe and effective potential treatments might be, speeding up the development of new therapies and reducing costs.

The research involves designing a computer-based platform using Artificial Intelligence (AI) and Machine Learning (ML). These advanced technologies will analyze existing data to simulate how DMD progresses and how patients might respond to treatments.

The outcomes of this research could transform how treatments for DMD are developed, ultimately improving the lives of individuals living with this condition