The meeting allowed participants to review the progress made, share emerging challenges, and define goals and strategies for the coming months.
The project’s innovative approach is based on a patient-centered perspective from its inception, integrating nine rare neurological diseases into a global collaborative model. The work spans from studying the natural history of the disease to defining molecular mechanisms, using patient-derived induced pluripotent stem cells. It also includes the conduct of clinical trials to gather clinical evidence and the proposal of new regulatory solutions, aiming to accelerate the approval of repurposed drugs for rare or ultra-rare diseases that currently have no approved therapeutic options.
This project has a five-year duration and is currently in its second year of activity.
SIMPATHIC is an acronym that reflects the project’s innovative purpose wich is “Accelerating drug repurposing for rare neurological, neurometabolic and neuromuscular disorders by exploiting SIMilarities in clinical and molecular PATHology.“
