The latest issue of Compass, our research publication, is now available online.
This summer 2015 issue includes information on our recent drug discovery grants, plus an update to our SMA drug pipeline.
Developing a treatment and cure for SMA is the driving force behind Cure SMA, and the goal of our three-stage research model.
Drug discovery is the pivotal second stage of that research model. The model begins with basic research, which investigates the causes and biology of SMA. Drug discovery then takes these basic research ideas and converts them into practical drug candidates that can be tested in the third stage: clinical trials.
Through this step-by-step model and through the diversity of therapeutic approaches we’ve identified, we are able to minimize the most common challenges of drug discovery by building a diverse “pipeline” of drug candidates. Fifteen years ago, we had just two potential drugs in the pipeline. Today we have eighteen, including seven now in clinical trials. Cure SMA has funded half of these drug programs.